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4min chapter
Rare Disease, Big Impact with Matt Wilsey
Raising Health
Advancements in Gene Therapy for NGLY1 Disorders
This chapter explores the innovative AV9 gene therapy, GS100, aimed at treating NGLY1 disorders. It highlights the treatment process, patient engagement, safety, clinical outcomes, and ethical concerns regarding equitable access to therapy across different age groups.
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Discover
highlights
Listen to the best highlights from the podcasts you love and dive into the full episode