In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone from zero to two, transforming the prospects for patients.
An FDA advisory committee has recommended approval of Zevra Therapeutics' arimoclomol for the ultra-rare disorder Niemann-Pick disease, three years after the regulator rejected the drug.
Join the conversation, on Tuesday 28th January @ 10 am ET - 3 pm GMT - 4 pm CET, in our webinar sponsored by Syneos Health, entitled ‘Health Trends 2025: Reimagining What's Possibl