The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.

The US Food and Drug Administration (FDA) has delayed its decision on Stealth BioTherapeutics’ new drug application (NDA) for elamipretide, a treatment for the ultra-rare disease Barth syndrome.

The FDA rejected the company’s initial NDA in 2021, citing the lack of an adequate trial to demonstrate efficacy.

Baylen Dupree, 22, stars in a new TLC series after she built a following on social media. She wants people to know what it's ...

Maharashtra has reported its first death due to Guillain-Barre Syndrome (GBS). A Pune-based Chartered Accountant was suffering from diarrhoea for a few days. Later, he could not move his hands and ...

A man in Maharashtra, who was suspected to have Guillain-Barre Syndrome (GBS) died in the Solapur district today while the number of cases of the immunological nerve disorder crossed 100 in Pune.

Initially, she dismissed the comments, but when medical professionals began weighing in, it led to her diagnosis of Cushing's syndrome.Jan. 24, 2025 ...

He dismisses my hurt feelings when his “teasing” about Trump Derangement Syndrome crosses the line. The final straw came last October, when I called to tell him I needed surgery. Before I ...

Naz Rahman has given his Buy rating due to a combination of factors surrounding Quoin Pharmaceuticals’ lead product, QRX003, which is currently in Phase 3 trials for Netherton Syndrome (NS).

Submission supported by positive results from global, Phase 3 4WHIM clinical trial; U.S. regulatory approval in WHIM syndrome granted in 2024 Decision on MAA expected in 1H 2026 BOSTON ...