Healio9y
Barth's 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.
thecardiologyadvisor.com2d
FDA Extends Review of Elamipretide for Barth Syndrome
The FDA has extended the review period for the New Drug Application for elamipretide for the treatment of Barth syndrome.
FierceBiotech7d
FDA delays decision on Stealth's 2nd shot at approval for ultrarare disease med
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 ...
UVA researchers develop MRI technique to predict heart disease
Researchers at UVA Health are taking a closer look at an often overlooked risk factor for heart disease: the fat surrounding your heart.
Morningstar7d
Stealth BioTherapeutics Announces PDUFA Action Date Extension for Elamipretide to Treat Patients with Barth Syndrome
About Barth Syndrome Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure ...
GlobalData on MSN3mon
FDA AdCom votes in favour of Stealth’s Barth syndrome drug
A US Food and Drug Administration (FDA) advisory committee (AdCom) has voted in support of Stealth Biotherapeutics’ ...
Monthly Prescribing Reference7d
FDA Extends Review of Elamipretide for Barth Syndrome
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 ...