SWARTZ CREEK, MI - Valentine’s Day was more than a celebration of love for Devin Addington—it was the day he received the ultimate gift: a new heart. The 20-year-old Swartz Creek resident who has ...
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GlobalData on MSNFDA AdCom votes in favour of Stealth’s Barth syndrome drugA US Food and Drug Administration (FDA) advisory committee (AdCom) has voted in support of Stealth Biotherapeutics’ ...
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.
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 ...
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 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 ...
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 ...
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