An ultra-rare disease drug missed on function but hit on survival and mortality. Is it enough for the FDA?

Max Gelman

Senior Editor

A California company developing a drug for an ultra-rare, universally fatal neurological disease manifesting in infancy reported Wednesday that its therapy missed the primary endpoint …

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An ultra-rare disease drug missed on function but hit on survival and mortality. Is it enough for the FDA?

Max Gelman

Senior Editor

Sign up to read this article for free.

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An ul­tra-rare dis­ease drug missed on func­tion but hit on sur­vival and mor­tal­i­ty. Is it enough for the FDA?

Max Gelman

Senior Editor

Sign up to read this article for free.

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An ultra-rare disease drug missed on function but hit on survival and mortality. Is it enough for the FDA?

FDA approves Lilly's Zepbound for sleep apnea, likely cracking open Medicare market

CagriSema’s weight loss data disappointed. What does Novo do next?

Three drugmakers sue US over 340B discounts to STD clinics they allege don’t qualify

Vertex gets 'vanza triple' approval and Trikafta expansion — with boxed warnings

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