Devin Addington was diagnosed with Barth syndrome at two years old, a rare genetic disorder that primarily affects boys.
Myeloid deficiency of the mitochondrial transacetylase tafazzin upregulates mtROS, disrupting UPR and calcium homeostasis, ...
A US Food and Drug Administration (FDA) advisory committee (AdCom) has voted in support of Stealth Biotherapeutics’ ...
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.
Barth’s syndrome in an inherited disorder in an X-linked fashion. The cardiac component includes dilated cardiomyopathy which can at times cause severe systolic congestive heart failure.
The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.
Barth syndrome (BTHS) is a rare genetic disorder primarily affecting males, characterized by a range of symptoms including cardiomyopathy, skeletal myopathy, and neutropenia. The condition is ...
Three months ago, the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 10-6 in support of elamipretide becoming the first approved treatment for Barth syndrome, an X-linked genetic ...
If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic, and the first FDA-approved therapy for Barth syndrome.
She is also a graduate of the Hearst Leadership Institute. Stacey currently serves as fundraising chairperson for the Barth Syndrome Foundation, a nonprofit advocacy organization that helps people ...
The new Prescription Drug User Fee Act target date is April 29, 2025. The Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for elamipretide for ...