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An FDA advisory committee agreed on Friday that Zevra Therapeutics had provided sufficient efficacy data supporting the approval of arimoclomol for Niemann-Pick disease type C.
Longeveron and Lexeo Therapeutics are working on CGT therapies to treat Alzheimer’s disease, but it’s not clear whether they have a better chance of success than traditional approaches.
The Genetic Metabolic Diseases Advisory Committee will meet on Friday to discuss Zevra’s modified scale to describe the efficacy of its drug candidate for the treatment of Niemann-Pick disease type C.
The FDA has four big events in the coming two weeks, including an advisory panel meeting for an ultra-rare disease.
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