FDA Rejects Rare Disease Treatment Despite Strong Clinical Data: Biohaven Challenges Regulatory Decision on Spinocerebellar Ataxia Drug

Biohaven Pharmaceuticals faced a significant setback when the U.S. Food and Drug Administration issued a Complete Response Letter (CRL) rejecting the company's New Drug Application for Vyglxia (troriluzole), a promising…

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Jim Wilson’s Pioneering Journey: Transforming Rare Disease Treatment Through Innovative Gene Therapy Part 2

Please go HERE to read Part 1 of this article The company, GEMMABIO, will pursue a number of rare, large indications.  A new spinout, RareTx, will be dedicated to Orphan…

Continue Reading Jim Wilson’s Pioneering Journey: Transforming Rare Disease Treatment Through Innovative Gene Therapy Part 2