Raising Health Rare Disease, Big Impact with Matt Wilsey
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Dec 10, 2024 Matt Wilsey, co-founder and CEO of Grace Science, shares a deeply personal journey after his daughter Grace was diagnosed with NGLY1 deficiency. He discusses the arduous path families face in rare disease diagnoses and the transformative advancements in genetic sequencing. Matt highlights innovative gene therapies, like AV9, aimed at treating NGLY1, and emphasizes the importance of strategic partnerships with scientists such as Nobel laureate Carolyn Bertozzi. The conversation also sheds light on navigating regulatory challenges in drug development.
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Grace's Arrival and the Promise
- Matt Wilsey's newborn daughter, Grace, faced a life-threatening medical emergency.
- This led him on a quest to diagnose and cure her rare disease, NGLY1 deficiency.
The Diagnostic Odyssey
- Diagnosing Grace's condition was a long and stressful journey for the Wilsey family.
- They consulted numerous specialists across the country before finding the root cause.
Celebrating Diagnosis
- Celebrate the milestone of receiving a diagnosis, as it marks a significant achievement.
- Early diagnosis can lead to timely interventions and better outcomes for patients and families.