The FDA has approved the first treatment for Niemann-Pick disease, type C, after years of advocacy and drug development efforts. The drug, arimoclomol, now marketed as Miplyffa, was a result of clinical trials a decade ago, driven by parents of children with the disease. The trials initially failed or had mixed results due to the varied symptoms of the disease, such as motor deficits, cognitive deficits, seizures, and difficulty hearing and swallowing. Niemann Pick disease progresses slowly, making it challenging to demonstrate the effectiveness of a drug, especially if the impact is minimal. The approval marks a significant milestone in the treatment of this rare neurological condition.
Source link
FDA approves first drug for rare neuron disease Niemann-Pick
