All articles tagged with #barth syndrome
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.
Stealth BioTherapeutics disclosed an FDA rejection letter to rally support for its experimental drug for Barth syndrome, a rare disease affecting about 150 people in the U.S., amid ongoing struggles to meet regulatory requirements and concerns over the company's future.
Stealth BioTherapeutics' drug for Barth syndrome, a rare illness affecting up to 150 people in the U.S., was unexpectedly rejected by the FDA, delaying access for patients and straining the company's finances, despite prior advisory committee support and ongoing discussions about new approval pathways.