The latest research report published by Vantage Market Research, titled "An Increase in Demand and Opportunities for the Global Barth Syndrome Treatment Market 2025-2035," provides a comprehensive ...

Patient engagement through advisory boards, surveys, and listening sessions can help address industry challenges like trial delays.

Treatments for dry eye disease, neuroendocrine tumors, chronic spontaneous urticaria, Barth syndrome, and migraine are under review.

Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in ...

The FDA is expected to decide on treatments for dry eye disease, neuroendocrine tumors, chronic spontaneous urticaria, Barth syndrome, and migraine. Reproxalap, a first-in-class small-molecule ...

A list of downloadable documents created during development.

Barth syndrome is caused by a mutation in the tafazzin gene, which results in reduced cardiolipin levels, a phospholipid that plays a major role in mitochondrial function. The disease is ...

根据PDUFA的预期目标日期，预计2025年4月，美国FDA将对4款创新药物的批准做出监管决定，本文将对这些疗法进行相关介绍。

But Devin was born with Barth Syndrome, a rare genetic condition that impacts 1 in 300,000 to 400,000 people. At nine weeks old, he underwent his first heart transplant. "When they did that ...