All articles tagged with #elamipretide
The FDA has granted accelerated approval to Forzinity (elamipretide) as the first treatment for Barth syndrome, a rare and serious mitochondrial disease, based on improvements in muscle strength that are likely to benefit patients, with further studies required to confirm these benefits.
Parents of children with Barth syndrome are urgently seeking FDA approval for the experimental drug elamipretide, which has shown promise in treating the rare disease but faces delays due to manufacturing issues and regulatory hurdles, risking the lives of affected children.
Patients with ultra-rare diseases like Hope Filchak's MLS syndrome face challenges in accessing treatments due to strict FDA approval processes, exemplified by the rejection of the drug elamipretide despite positive advisory committee recommendations, raising concerns about the balance between safety and urgent patient needs.