The Readout Loud 362: A mother’s perspective on Sarepta’s gene therapy halt
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Jul 24, 2025 Kaylin West, mother of 7-year-old Cooper with Duchenne muscular dystrophy, shares her powerful insights on Sarepta's decision to halt its gene therapy, Elevidys. She reflects on her family's journey through the hopeful yet tumultuous world of gene treatments. The podcast also delves into the emotional impact of such medical developments, highlighting the importance of community support for families navigating similar challenges. Alongside biotech innovations and new drug developments, Kaylin's personal story adds a poignant depth to the discussion.
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Orexin Drugs for Wakefulness
- Drugs mimicking orexin neurons show promise for narcolepsy type 1 by improving wakefulness dramatically.
- This new drug class could extend to broader conditions like depression and Alzheimer's where daytime sleepiness is a symptom.
Decline in Oncology Financing
- Oncology financing is shrinking with fewer startups raising significant investment this year.
- Oversaturation and shifting investor priorities dampen enthusiasm for new cancer ventures.
Sarepta's Gene Therapy Crisis
- Sarepta's Levitis gene therapy was halted by the FDA after multiple patient deaths from liver failure.
- The therapy faces an uncertain future as FDA officials doubt Sarepta can demonstrate safe use with new immunosuppression protocols.