"That arc – her rise and her decline – became the essence of Violet’s story."
Foreward
This is Part II of Violet’s story through The Portrait Project, told by her father, David. To meet the bright, beautiful little girl at the heart of it all – and to learn about the hope that carried Violet's family through a clinical trial — read Part I.
Part II
“You know those movies with a twist ending? Where everything you thought was going to happen takes a completely unexpected turn? I’m a doctor, but I’m also a filmmaker, so I think a lot about narrative as I move through life. I had to reconcile with the fact that life doesn’t always follow the arc you’d expect. You might imagine yourself as the hero who saves the world, but it’s not always like that. Sometimes it’s dangerous. And in our case, devastating. It was a strange experience, watching as my son was growing and hitting milestones, while Violet’s milestones stopped. It felt like I was watching her grow up, only to nurse her right back down. That arc – her rise and her decline – became the essence of Violet’s story.
Violet’s passing was sudden. It wasn’t because of the medication from the trial, but a respiratory issue typical in this population. Sometimes I wonder if it would’ve been better to keep her in the hospital longer. Or if we should’ve brought in more support, like home nursing or hospice care. But when you’re in the middle of it, you’re not thinking about yourself. You’re just trying to get through the day. And in all honesty, we were burned out from caretaking and running on empty. Hindsight is always 20/20. So it happened the way it happened. And all of it meant something. The trial gave us hope, and Violet’s contributions to science mattered. The blood tests, the lumbar punctures, all of it. It’s wild to think her clinical trial was originally for Tay-Sachs disease – not even GM1. GM1 kids like Violet were the secondary population. The program was scrapped, since the Tay-Sachs group didn’t show benefit. But for the GM1 group, some of us saw positive results. So we’re still advocating for it.
I saw a quote from a bereaved parent recently. They were asked, “Would you do it again, knowing what you’d have to go through?” And their answer was yes, absolutely. And I agree. Because despite how hard the pain was and still is, there were moments of peace. The quiet moments when Violet was stable, and things felt okay. Those moments were beautiful, and they made it all worth it.
“Violet’s essence is scattered all over California.”
When Violet died, I knew I had to process it all. But I couldn’t remember much. I barely even remember when we did Part I of this story back in 2023. It’s all a blur. We were in survival mode. I think I was also just so focused on how tragic and painful her death was, and rightfully so. But over time I’ve realized this experience was so much more than that. Violet’s time with us was short, but it was meaningful. It changed the course of our lives. I’ve started to piece together memories of those days, and certain moments stand out. When a child is in a condition like Violet’s, all the usual ways of communicating go away. So we had to learn new ways to connect. Simple things, like holding her close, just to make sure she felt us there. Those small gestures became our language. She couldn’t move much, but still absorbed everything around her. She recognized our faces, our voices, our laughter. Those are the moments that stick with me now.
And the fact that we got her into a clinical trial – against all odds – was incredible. We’re grateful for it. I know that the trial medication seemed to have a positive impact. Violet was more active, she went to school for the first time, and she finished a year of preschool. Maybe the medication didn’t have a lasting effect – or maybe she just adjusted to it – but there were these little moments where she sat up, watched cartoons, or clapped. Those moments were incredible. And because of Violet, my wife and I, who are both physicians, became deeply involved in medical advocacy, fighting for rare diseases.
When Violet was alive, people were deeply moved by her story. After she passed, we wanted to make sure she was remembered. Our friends in film and gaming have helped us do that. One credited her as an executive producer on an upcoming animated movie called ‘SNEAKS.’ Another ended their video game with “In Memory of Violet.” There’s also a plaque at the top of Signal Hill in Long Beach, donated by friends in her memory. In a way, Violet’s essence is scattered all over California. It’s beautiful.
“I feel her energy guiding me.”
It’s been about a year and a half since she passed. We’re doing okay. Our son is healthy, running around, thriving. We’re moving forward. But our advocacy doesn’t stop. As daunting as it is – especially given the current funding and healthcare landscape – the only way to advance science and therapies is through clinical trials. And that requires advocacy. I’ve met many bereaved parents in this community. Some keep the flame of advocacy alive. Others step back. They might donate, offer kind words, and then eventually they move on. I’ve found that the energy Violet had in her life is still with me. Every time I do something – whether it’s through the CureGM1 Foundation or going to the Senate – I feel her energy guiding me. That’s why I continue. Even after she was gone, the drive to keep going never left.
In the year that followed Violet’s death, everything felt connected to her and to the advocacy that carries on in her name. One of the most meaningful moments was being invited back to the California State Senate floor by Senator Padilla, for GM1 Gangliosidosis Awareness Day in 2024. Unlike a governor-declared day, GM1 Day is recognized through a Senate concurrent resolution. It requires a vote and approval by both the Senate and the House, year after year. It carries real weight. I really thought it would be just a one-time thing, back in 2023 when we first helped establish the day. But Senator Padilla, who represents San Diego, remembered us. His office has continued to invite us back, year after year, to sponsor the resolution. I’m grateful for that. Our entire family was able to attend, which made it even more special. We were invited back again this year, too. It’s an opportunity to raise awareness and bring my family along for another unforgettable experience honoring Violet.
We’re also launching a new initiative with the CureGM1 Foundation. We invested in a cell line that will serve as the foundation for developing enzyme replacement therapy for GM1. The science behind it is straightforward: GM1 is caused by the absence of a crucial enzyme, and without that enzyme, spinal cord nerves can’t regenerate. But if we can replace it - which has worked for other diseases - we may be able to restore function. It’s promising.
Working with the foundation brings me joy and gives me hope, especially when I consider the traction we’ve made in just a few years. Last August I was unexpectedly honored with Sanofi’s 2024 TORCH Award for advocacy. I had the surreal opportunity to meet and personally thank the researchers who created the clinical trial treatment that helped Violet. I told them it made a real difference for her, and that it brought some light to our family during a very dark time. This medication is now available to GM1 kids through compassionate access. It’s not a cure, but it’s a step forward. Kids in Europe are already receiving it. So it’s incredibly encouraging.
“For our generation, storytelling is the language we use to process and connect.”
Our experience in the rare disease world reignited an old passion for storytelling and filmmaking, which started for me back in college. During the summers, I’d go back home to LA and spend time with friends at USC’s film school, helping out on their projects. It was such a cool learning experience, but I always had the itch to make something of my own. Eventually I did. I made a short film called ‘Code,’ about a medical resident on a 24-hour call shift who’s grappling with burnout after a close friend dies by suicide. We made it in 2018, pre-pandemic and before burnout and mental health were the widespread conversations they are today. That short got into some festivals, won a few local awards, and things started gaining momentum. I made another film in 2019 called The Uppermost, which won Best Film at the Utah Film Awards that April. Our little production company was really starting to take off. Then, of course, the pandemic hit and everything came to a halt. Filmmaking got lost in the haze of what came next. I put it on hold for a while, for obvious reasons. But lately I’ve been feeling the pull again. I think I’m ready to dive back in.
For some families, it’s too painful to talk about what they’ve been through. They receive the diagnosis and retreat inward. I get it. The idea of advocacy, or publicly sharing something so personal, can feel impossible. And in some communities – especially among older generations – it’s culturally discouraged. You just don’t talk about these things. Even my own parents don’t always understand what we’re doing. They support us in their own way, but they’ll ask questions like, “Has this brought you a cure?” or “What’s the point of doing all of this?” It’s hard for them to see value in something that doesn’t yield immediate, tangible results. I’ve had to gently challenge their way of thinking, because often the most meaningful work doesn’t come with a cure or a paycheck – it comes with connection and with purpose. Sharing our story has allowed us to connect with others, and to advocate for families navigating similar heartbreak. Storytelling became part of our healing process. I think for our generation, storytelling is the language we use to process and connect. It’s not something to hide from. It can be transformative. And I think that’s something worth leaning into.
“Violet inspired passion.”
You asked me earlier how I want Violet to be remembered. The answer is that Violet inspired passion. She still does, and she always will. She inspires so much love and drive in everyone she touches. It’s amazing to witness, even now.
Back in college, I had big dreams. I wanted to be the best doctor in the world, make an Oscar-winning film, and accomplish everything I could. But life has a way of beating you down. Reality sets in, responsibilities pile up, and somewhere along the way, you lose some of that drive. But Violet’s story and presence helped many of us rediscover it – the drive to keep going and to live fully, even in the face of loss. For a lot of people, Violet’s life and death was a reawakening. And it’s crazy how it all unfolded when I turned 40, right in the middle of my life. It’s been a wild transition into this second half. It took me a while to grow up. But now, looking back, I can see what a turning point it’s been. Violet changed everything."
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Foreward
This is Part II of Violet’s story through The Portrait Project, told by her father, David. To meet the bright, beautiful little girl at the heart of it all – and to learn about the hope that carried Violet's family through a clinical trial — read Part I.
Part II
“You know those movies with a twist ending? Where everything you thought was going to happen takes a completely unexpected turn? I’m a doctor, but I’m also a filmmaker, so I think a lot about narrative as I move through life. I had to reconcile with the fact that life doesn’t always follow the arc you’d expect. You might imagine yourself as the hero who saves the world, but it’s not always like that. Sometimes it’s dangerous. And in our case, devastating. It was a strange experience, watching as my son was growing and hitting milestones, while Violet’s milestones stopped. It felt like I was watching her grow up, only to nurse her right back down. That arc – her rise and her decline – became the essence of Violet’s story.
Violet’s passing was sudden. It wasn’t because of the medication from the trial, but a respiratory issue typical in this population. Sometimes I wonder if it would’ve been better to keep her in the hospital longer. Or if we should’ve brought in more support, like home nursing or hospice care. But when you’re in the middle of it, you’re not thinking about yourself. You’re just trying to get through the day. And in all honesty, we were burned out from caretaking and running on empty. Hindsight is always 20/20. So it happened the way it happened. And all of it meant something. The trial gave us hope, and Violet’s contributions to science mattered. The blood tests, the lumbar punctures, all of it. It’s wild to think her clinical trial was originally for Tay-Sachs disease – not even GM1. GM1 kids like Violet were the secondary population. The program was scrapped, since the Tay-Sachs group didn’t show benefit. But for the GM1 group, some of us saw positive results. So we’re still advocating for it.
I saw a quote from a bereaved parent recently. They were asked, “Would you do it again, knowing what you’d have to go through?” And their answer was yes, absolutely. And I agree. Because despite how hard the pain was and still is, there were moments of peace. The quiet moments when Violet was stable, and things felt okay. Those moments were beautiful, and they made it all worth it.
“Violet’s essence is scattered all over California.”
When Violet died, I knew I had to process it all. But I couldn’t remember much. I barely even remember when we did Part I of this story back in 2023. It’s all a blur. We were in survival mode. I think I was also just so focused on how tragic and painful her death was, and rightfully so. But over time I’ve realized this experience was so much more than that. Violet’s time with us was short, but it was meaningful. It changed the course of our lives. I’ve started to piece together memories of those days, and certain moments stand out. When a child is in a condition like Violet’s, all the usual ways of communicating go away. So we had to learn new ways to connect. Simple things, like holding her close, just to make sure she felt us there. Those small gestures became our language. She couldn’t move much, but still absorbed everything around her. She recognized our faces, our voices, our laughter. Those are the moments that stick with me now.
And the fact that we got her into a clinical trial – against all odds – was incredible. We’re grateful for it. I know that the trial medication seemed to have a positive impact. Violet was more active, she went to school for the first time, and she finished a year of preschool. Maybe the medication didn’t have a lasting effect – or maybe she just adjusted to it – but there were these little moments where she sat up, watched cartoons, or clapped. Those moments were incredible. And because of Violet, my wife and I, who are both physicians, became deeply involved in medical advocacy, fighting for rare diseases.
When Violet was alive, people were deeply moved by her story. After she passed, we wanted to make sure she was remembered. Our friends in film and gaming have helped us do that. One credited her as an executive producer on an upcoming animated movie called ‘SNEAKS.’ Another ended their video game with “In Memory of Violet.” There’s also a plaque at the top of Signal Hill in Long Beach, donated by friends in her memory. In a way, Violet’s essence is scattered all over California. It’s beautiful.
“I feel her energy guiding me.”
It’s been about a year and a half since she passed. We’re doing okay. Our son is healthy, running around, thriving. We’re moving forward. But our advocacy doesn’t stop. As daunting as it is – especially given the current funding and healthcare landscape – the only way to advance science and therapies is through clinical trials. And that requires advocacy. I’ve met many bereaved parents in this community. Some keep the flame of advocacy alive. Others step back. They might donate, offer kind words, and then eventually they move on. I’ve found that the energy Violet had in her life is still with me. Every time I do something – whether it’s through the CureGM1 Foundation or going to the Senate – I feel her energy guiding me. That’s why I continue. Even after she was gone, the drive to keep going never left.
In the year that followed Violet’s death, everything felt connected to her and to the advocacy that carries on in her name. One of the most meaningful moments was being invited back to the California State Senate floor by Senator Padilla, for GM1 Gangliosidosis Awareness Day in 2024. Unlike a governor-declared day, GM1 Day is recognized through a Senate concurrent resolution. It requires a vote and approval by both the Senate and the House, year after year. It carries real weight. I really thought it would be just a one-time thing, back in 2023 when we first helped establish the day. But Senator Padilla, who represents San Diego, remembered us. His office has continued to invite us back, year after year, to sponsor the resolution. I’m grateful for that. Our entire family was able to attend, which made it even more special. We were invited back again this year, too. It’s an opportunity to raise awareness and bring my family along for another unforgettable experience honoring Violet.
We’re also launching a new initiative with the CureGM1 Foundation. We invested in a cell line that will serve as the foundation for developing enzyme replacement therapy for GM1. The science behind it is straightforward: GM1 is caused by the absence of a crucial enzyme, and without that enzyme, spinal cord nerves can’t regenerate. But if we can replace it - which has worked for other diseases - we may be able to restore function. It’s promising.
Working with the foundation brings me joy and gives me hope, especially when I consider the traction we’ve made in just a few years. Last August I was unexpectedly honored with Sanofi’s 2024 TORCH Award for advocacy. I had the surreal opportunity to meet and personally thank the researchers who created the clinical trial treatment that helped Violet. I told them it made a real difference for her, and that it brought some light to our family during a very dark time. This medication is now available to GM1 kids through compassionate access. It’s not a cure, but it’s a step forward. Kids in Europe are already receiving it. So it’s incredibly encouraging.
“For our generation, storytelling is the language we use to process and connect.”
Our experience in the rare disease world reignited an old passion for storytelling and filmmaking, which started for me back in college. During the summers, I’d go back home to LA and spend time with friends at USC’s film school, helping out on their projects. It was such a cool learning experience, but I always had the itch to make something of my own. Eventually I did. I made a short film called ‘Code,’ about a medical resident on a 24-hour call shift who’s grappling with burnout after a close friend dies by suicide. We made it in 2018, pre-pandemic and before burnout and mental health were the widespread conversations they are today. That short got into some festivals, won a few local awards, and things started gaining momentum. I made another film in 2019 called The Uppermost, which won Best Film at the Utah Film Awards that April. Our little production company was really starting to take off. Then, of course, the pandemic hit and everything came to a halt. Filmmaking got lost in the haze of what came next. I put it on hold for a while, for obvious reasons. But lately I’ve been feeling the pull again. I think I’m ready to dive back in.
For some families, it’s too painful to talk about what they’ve been through. They receive the diagnosis and retreat inward. I get it. The idea of advocacy, or publicly sharing something so personal, can feel impossible. And in some communities – especially among older generations – it’s culturally discouraged. You just don’t talk about these things. Even my own parents don’t always understand what we’re doing. They support us in their own way, but they’ll ask questions like, “Has this brought you a cure?” or “What’s the point of doing all of this?” It’s hard for them to see value in something that doesn’t yield immediate, tangible results. I’ve had to gently challenge their way of thinking, because often the most meaningful work doesn’t come with a cure or a paycheck – it comes with connection and with purpose. Sharing our story has allowed us to connect with others, and to advocate for families navigating similar heartbreak. Storytelling became part of our healing process. I think for our generation, storytelling is the language we use to process and connect. It’s not something to hide from. It can be transformative. And I think that’s something worth leaning into.
“Violet inspired passion.”
You asked me earlier how I want Violet to be remembered. The answer is that Violet inspired passion. She still does, and she always will. She inspires so much love and drive in everyone she touches. It’s amazing to witness, even now.
Back in college, I had big dreams. I wanted to be the best doctor in the world, make an Oscar-winning film, and accomplish everything I could. But life has a way of beating you down. Reality sets in, responsibilities pile up, and somewhere along the way, you lose some of that drive. But Violet’s story and presence helped many of us rediscover it – the drive to keep going and to live fully, even in the face of loss. For a lot of people, Violet’s life and death was a reawakening. And it’s crazy how it all unfolded when I turned 40, right in the middle of my life. It’s been a wild transition into this second half. It took me a while to grow up. But now, looking back, I can see what a turning point it’s been. Violet changed everything."
Foreward
This is Part II of Violet’s story through The Portrait Project, told by her father, David. To meet the bright, beautiful little girl at the heart of it all – and to learn about the hope that carried Violet's family through a clinical trial — read Part I.
Part II
“You know those movies with a twist ending? Where everything you thought was going to happen takes a completely unexpected turn? I’m a doctor, but I’m also a filmmaker, so I think a lot about narrative as I move through life. I had to reconcile with the fact that life doesn’t always follow the arc you’d expect. You might imagine yourself as the hero who saves the world, but it’s not always like that. Sometimes it’s dangerous. And in our case, devastating. It was a strange experience, watching as my son was growing and hitting milestones, while Violet’s milestones stopped. It felt like I was watching her grow up, only to nurse her right back down. That arc – her rise and her decline – became the essence of Violet’s story.
Violet’s passing was sudden. It wasn’t because of the medication from the trial, but a respiratory issue typical in this population. Sometimes I wonder if it would’ve been better to keep her in the hospital longer. Or if we should’ve brought in more support, like home nursing or hospice care. But when you’re in the middle of it, you’re not thinking about yourself. You’re just trying to get through the day. And in all honesty, we were burned out from caretaking and running on empty. Hindsight is always 20/20. So it happened the way it happened. And all of it meant something. The trial gave us hope, and Violet’s contributions to science mattered. The blood tests, the lumbar punctures, all of it. It’s wild to think her clinical trial was originally for Tay-Sachs disease – not even GM1. GM1 kids like Violet were the secondary population. The program was scrapped, since the Tay-Sachs group didn’t show benefit. But for the GM1 group, some of us saw positive results. So we’re still advocating for it.
I saw a quote from a bereaved parent recently. They were asked, “Would you do it again, knowing what you’d have to go through?” And their answer was yes, absolutely. And I agree. Because despite how hard the pain was and still is, there were moments of peace. The quiet moments when Violet was stable, and things felt okay. Those moments were beautiful, and they made it all worth it.
“Violet’s essence is scattered all over California.”
When Violet died, I knew I had to process it all. But I couldn’t remember much. I barely even remember when we did Part I of this story back in 2023. It’s all a blur. We were in survival mode. I think I was also just so focused on how tragic and painful her death was, and rightfully so. But over time I’ve realized this experience was so much more than that. Violet’s time with us was short, but it was meaningful. It changed the course of our lives. I’ve started to piece together memories of those days, and certain moments stand out. When a child is in a condition like Violet’s, all the usual ways of communicating go away. So we had to learn new ways to connect. Simple things, like holding her close, just to make sure she felt us there. Those small gestures became our language. She couldn’t move much, but still absorbed everything around her. She recognized our faces, our voices, our laughter. Those are the moments that stick with me now.
And the fact that we got her into a clinical trial – against all odds – was incredible. We’re grateful for it. I know that the trial medication seemed to have a positive impact. Violet was more active, she went to school for the first time, and she finished a year of preschool. Maybe the medication didn’t have a lasting effect – or maybe she just adjusted to it – but there were these little moments where she sat up, watched cartoons, or clapped. Those moments were incredible. And because of Violet, my wife and I, who are both physicians, became deeply involved in medical advocacy, fighting for rare diseases.
When Violet was alive, people were deeply moved by her story. After she passed, we wanted to make sure she was remembered. Our friends in film and gaming have helped us do that. One credited her as an executive producer on an upcoming animated movie called ‘SNEAKS.’ Another ended their video game with “In Memory of Violet.” There’s also a plaque at the top of Signal Hill in Long Beach, donated by friends in her memory. In a way, Violet’s essence is scattered all over California. It’s beautiful.
“I feel her energy guiding me.”
It’s been about a year and a half since she passed. We’re doing okay. Our son is healthy, running around, thriving. We’re moving forward. But our advocacy doesn’t stop. As daunting as it is – especially given the current funding and healthcare landscape – the only way to advance science and therapies is through clinical trials. And that requires advocacy. I’ve met many bereaved parents in this community. Some keep the flame of advocacy alive. Others step back. They might donate, offer kind words, and then eventually they move on. I’ve found that the energy Violet had in her life is still with me. Every time I do something – whether it’s through the CureGM1 Foundation or going to the Senate – I feel her energy guiding me. That’s why I continue. Even after she was gone, the drive to keep going never left.
In the year that followed Violet’s death, everything felt connected to her and to the advocacy that carries on in her name. One of the most meaningful moments was being invited back to the California State Senate floor by Senator Padilla, for GM1 Gangliosidosis Awareness Day in 2024. Unlike a governor-declared day, GM1 Day is recognized through a Senate concurrent resolution. It requires a vote and approval by both the Senate and the House, year after year. It carries real weight. I really thought it would be just a one-time thing, back in 2023 when we first helped establish the day. But Senator Padilla, who represents San Diego, remembered us. His office has continued to invite us back, year after year, to sponsor the resolution. I’m grateful for that. Our entire family was able to attend, which made it even more special. We were invited back again this year, too. It’s an opportunity to raise awareness and bring my family along for another unforgettable experience honoring Violet.
We’re also launching a new initiative with the CureGM1 Foundation. We invested in a cell line that will serve as the foundation for developing enzyme replacement therapy for GM1. The science behind it is straightforward: GM1 is caused by the absence of a crucial enzyme, and without that enzyme, spinal cord nerves can’t regenerate. But if we can replace it - which has worked for other diseases - we may be able to restore function. It’s promising.
Working with the foundation brings me joy and gives me hope, especially when I consider the traction we’ve made in just a few years. Last August I was unexpectedly honored with Sanofi’s 2024 TORCH Award for advocacy. I had the surreal opportunity to meet and personally thank the researchers who created the clinical trial treatment that helped Violet. I told them it made a real difference for her, and that it brought some light to our family during a very dark time. This medication is now available to GM1 kids through compassionate access. It’s not a cure, but it’s a step forward. Kids in Europe are already receiving it. So it’s incredibly encouraging.
“For our generation, storytelling is the language we use to process and connect.”
Our experience in the rare disease world reignited an old passion for storytelling and filmmaking, which started for me back in college. During the summers, I’d go back home to LA and spend time with friends at USC’s film school, helping out on their projects. It was such a cool learning experience, but I always had the itch to make something of my own. Eventually I did. I made a short film called ‘Code,’ about a medical resident on a 24-hour call shift who’s grappling with burnout after a close friend dies by suicide. We made it in 2018, pre-pandemic and before burnout and mental health were the widespread conversations they are today. That short got into some festivals, won a few local awards, and things started gaining momentum. I made another film in 2019 called The Uppermost, which won Best Film at the Utah Film Awards that April. Our little production company was really starting to take off. Then, of course, the pandemic hit and everything came to a halt. Filmmaking got lost in the haze of what came next. I put it on hold for a while, for obvious reasons. But lately I’ve been feeling the pull again. I think I’m ready to dive back in.
For some families, it’s too painful to talk about what they’ve been through. They receive the diagnosis and retreat inward. I get it. The idea of advocacy, or publicly sharing something so personal, can feel impossible. And in some communities – especially among older generations – it’s culturally discouraged. You just don’t talk about these things. Even my own parents don’t always understand what we’re doing. They support us in their own way, but they’ll ask questions like, “Has this brought you a cure?” or “What’s the point of doing all of this?” It’s hard for them to see value in something that doesn’t yield immediate, tangible results. I’ve had to gently challenge their way of thinking, because often the most meaningful work doesn’t come with a cure or a paycheck – it comes with connection and with purpose. Sharing our story has allowed us to connect with others, and to advocate for families navigating similar heartbreak. Storytelling became part of our healing process. I think for our generation, storytelling is the language we use to process and connect. It’s not something to hide from. It can be transformative. And I think that’s something worth leaning into.
“Violet inspired passion.”
You asked me earlier how I want Violet to be remembered. The answer is that Violet inspired passion. She still does, and she always will. She inspires so much love and drive in everyone she touches. It’s amazing to witness, even now.
Back in college, I had big dreams. I wanted to be the best doctor in the world, make an Oscar-winning film, and accomplish everything I could. But life has a way of beating you down. Reality sets in, responsibilities pile up, and somewhere along the way, you lose some of that drive. But Violet’s story and presence helped many of us rediscover it – the drive to keep going and to live fully, even in the face of loss. For a lot of people, Violet’s life and death was a reawakening. And it’s crazy how it all unfolded when I turned 40, right in the middle of my life. It’s been a wild transition into this second half. It took me a while to grow up. But now, looking back, I can see what a turning point it’s been. Violet changed everything."
Foreward
This is Part II of Violet’s story through The Portrait Project, told by her father, David. To meet the bright, beautiful little girl at the heart of it all – and to learn about the hope that carried Violet's family through a clinical trial — read Part I.
Part II
“You know those movies with a twist ending? Where everything you thought was going to happen takes a completely unexpected turn? I’m a doctor, but I’m also a filmmaker, so I think a lot about narrative as I move through life. I had to reconcile with the fact that life doesn’t always follow the arc you’d expect. You might imagine yourself as the hero who saves the world, but it’s not always like that. Sometimes it’s dangerous. And in our case, devastating. It was a strange experience, watching as my son was growing and hitting milestones, while Violet’s milestones stopped. It felt like I was watching her grow up, only to nurse her right back down. That arc – her rise and her decline – became the essence of Violet’s story.
Violet’s passing was sudden. It wasn’t because of the medication from the trial, but a respiratory issue typical in this population. Sometimes I wonder if it would’ve been better to keep her in the hospital longer. Or if we should’ve brought in more support, like home nursing or hospice care. But when you’re in the middle of it, you’re not thinking about yourself. You’re just trying to get through the day. And in all honesty, we were burned out from caretaking and running on empty. Hindsight is always 20/20. So it happened the way it happened. And all of it meant something. The trial gave us hope, and Violet’s contributions to science mattered. The blood tests, the lumbar punctures, all of it. It’s wild to think her clinical trial was originally for Tay-Sachs disease – not even GM1. GM1 kids like Violet were the secondary population. The program was scrapped, since the Tay-Sachs group didn’t show benefit. But for the GM1 group, some of us saw positive results. So we’re still advocating for it.
I saw a quote from a bereaved parent recently. They were asked, “Would you do it again, knowing what you’d have to go through?” And their answer was yes, absolutely. And I agree. Because despite how hard the pain was and still is, there were moments of peace. The quiet moments when Violet was stable, and things felt okay. Those moments were beautiful, and they made it all worth it.
“Violet’s essence is scattered all over California.”
When Violet died, I knew I had to process it all. But I couldn’t remember much. I barely even remember when we did Part I of this story back in 2023. It’s all a blur. We were in survival mode. I think I was also just so focused on how tragic and painful her death was, and rightfully so. But over time I’ve realized this experience was so much more than that. Violet’s time with us was short, but it was meaningful. It changed the course of our lives. I’ve started to piece together memories of those days, and certain moments stand out. When a child is in a condition like Violet’s, all the usual ways of communicating go away. So we had to learn new ways to connect. Simple things, like holding her close, just to make sure she felt us there. Those small gestures became our language. She couldn’t move much, but still absorbed everything around her. She recognized our faces, our voices, our laughter. Those are the moments that stick with me now.
And the fact that we got her into a clinical trial – against all odds – was incredible. We’re grateful for it. I know that the trial medication seemed to have a positive impact. Violet was more active, she went to school for the first time, and she finished a year of preschool. Maybe the medication didn’t have a lasting effect – or maybe she just adjusted to it – but there were these little moments where she sat up, watched cartoons, or clapped. Those moments were incredible. And because of Violet, my wife and I, who are both physicians, became deeply involved in medical advocacy, fighting for rare diseases.
When Violet was alive, people were deeply moved by her story. After she passed, we wanted to make sure she was remembered. Our friends in film and gaming have helped us do that. One credited her as an executive producer on an upcoming animated movie called ‘SNEAKS.’ Another ended their video game with “In Memory of Violet.” There’s also a plaque at the top of Signal Hill in Long Beach, donated by friends in her memory. In a way, Violet’s essence is scattered all over California. It’s beautiful.
“I feel her energy guiding me.”
It’s been about a year and a half since she passed. We’re doing okay. Our son is healthy, running around, thriving. We’re moving forward. But our advocacy doesn’t stop. As daunting as it is – especially given the current funding and healthcare landscape – the only way to advance science and therapies is through clinical trials. And that requires advocacy. I’ve met many bereaved parents in this community. Some keep the flame of advocacy alive. Others step back. They might donate, offer kind words, and then eventually they move on. I’ve found that the energy Violet had in her life is still with me. Every time I do something – whether it’s through the CureGM1 Foundation or going to the Senate – I feel her energy guiding me. That’s why I continue. Even after she was gone, the drive to keep going never left.
In the year that followed Violet’s death, everything felt connected to her and to the advocacy that carries on in her name. One of the most meaningful moments was being invited back to the California State Senate floor by Senator Padilla, for GM1 Gangliosidosis Awareness Day in 2024. Unlike a governor-declared day, GM1 Day is recognized through a Senate concurrent resolution. It requires a vote and approval by both the Senate and the House, year after year. It carries real weight. I really thought it would be just a one-time thing, back in 2023 when we first helped establish the day. But Senator Padilla, who represents San Diego, remembered us. His office has continued to invite us back, year after year, to sponsor the resolution. I’m grateful for that. Our entire family was able to attend, which made it even more special. We were invited back again this year, too. It’s an opportunity to raise awareness and bring my family along for another unforgettable experience honoring Violet.
We’re also launching a new initiative with the CureGM1 Foundation. We invested in a cell line that will serve as the foundation for developing enzyme replacement therapy for GM1. The science behind it is straightforward: GM1 is caused by the absence of a crucial enzyme, and without that enzyme, spinal cord nerves can’t regenerate. But if we can replace it - which has worked for other diseases - we may be able to restore function. It’s promising.
Working with the foundation brings me joy and gives me hope, especially when I consider the traction we’ve made in just a few years. Last August I was unexpectedly honored with Sanofi’s 2024 TORCH Award for advocacy. I had the surreal opportunity to meet and personally thank the researchers who created the clinical trial treatment that helped Violet. I told them it made a real difference for her, and that it brought some light to our family during a very dark time. This medication is now available to GM1 kids through compassionate access. It’s not a cure, but it’s a step forward. Kids in Europe are already receiving it. So it’s incredibly encouraging.
“For our generation, storytelling is the language we use to process and connect.”
Our experience in the rare disease world reignited an old passion for storytelling and filmmaking, which started for me back in college. During the summers, I’d go back home to LA and spend time with friends at USC’s film school, helping out on their projects. It was such a cool learning experience, but I always had the itch to make something of my own. Eventually I did. I made a short film called ‘Code,’ about a medical resident on a 24-hour call shift who’s grappling with burnout after a close friend dies by suicide. We made it in 2018, pre-pandemic and before burnout and mental health were the widespread conversations they are today. That short got into some festivals, won a few local awards, and things started gaining momentum. I made another film in 2019 called The Uppermost, which won Best Film at the Utah Film Awards that April. Our little production company was really starting to take off. Then, of course, the pandemic hit and everything came to a halt. Filmmaking got lost in the haze of what came next. I put it on hold for a while, for obvious reasons. But lately I’ve been feeling the pull again. I think I’m ready to dive back in.
For some families, it’s too painful to talk about what they’ve been through. They receive the diagnosis and retreat inward. I get it. The idea of advocacy, or publicly sharing something so personal, can feel impossible. And in some communities – especially among older generations – it’s culturally discouraged. You just don’t talk about these things. Even my own parents don’t always understand what we’re doing. They support us in their own way, but they’ll ask questions like, “Has this brought you a cure?” or “What’s the point of doing all of this?” It’s hard for them to see value in something that doesn’t yield immediate, tangible results. I’ve had to gently challenge their way of thinking, because often the most meaningful work doesn’t come with a cure or a paycheck – it comes with connection and with purpose. Sharing our story has allowed us to connect with others, and to advocate for families navigating similar heartbreak. Storytelling became part of our healing process. I think for our generation, storytelling is the language we use to process and connect. It’s not something to hide from. It can be transformative. And I think that’s something worth leaning into.
“Violet inspired passion.”
You asked me earlier how I want Violet to be remembered. The answer is that Violet inspired passion. She still does, and she always will. She inspires so much love and drive in everyone she touches. It’s amazing to witness, even now.
Back in college, I had big dreams. I wanted to be the best doctor in the world, make an Oscar-winning film, and accomplish everything I could. But life has a way of beating you down. Reality sets in, responsibilities pile up, and somewhere along the way, you lose some of that drive. But Violet’s story and presence helped many of us rediscover it – the drive to keep going and to live fully, even in the face of loss. For a lot of people, Violet’s life and death was a reawakening. And it’s crazy how it all unfolded when I turned 40, right in the middle of my life. It’s been a wild transition into this second half. It took me a while to grow up. But now, looking back, I can see what a turning point it’s been. Violet changed everything."
Representation Matters
Increasing diversity in clinical trials builds trust, promotes health equity, and leads to more effective treatments and better outcomes (NEJM). But there is much work to be done - and barriers to break - to improve awareness and access for all people.
Do you know someone who is a member of a marginalized community who has participated in a clinical trial? If so, we’d love to meet them and share their story. We hope to represent the many faces of clinical trials through this project, and inspire others by shining a light on their experience.
They can contact us here.
