GlobalData on MSN6d
Stealth faces another hurdle as FDA delays decision on Barth syndrome drug
The FDA rejected the company’s initial NDA in 2021, citing the lack of an adequate trial to demonstrate efficacy.
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.
Yahoo Finance6d
Stealth faces another hurdle as FDA delays decision on Barth syndrome drug
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.
Nature6d
Barth Syndrome and Mitochondrial Cardiolipin Metabolism
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 ...
ENDPOINTS NEWS6d
FDA de­lays de­ci­sion on Stealth’s Barth syn­drome drug; Tar­getRx rais­es $50M
FDA delays Stealth BioTherapeutics' elamipretide decision to April 29; Valneva's Ixchiq succeeds in child safety trial; TargetRx raises $50M; Ab&B Bio-Tech files for HKEX IPO ...
The Pharma Letter6d
FDA delays PDUFA action date for elamipretide
US clinical-stage drug developer Stealth BioTherapeutics announced that the US Food and Drug Administration (FDA) has ...
Phys.org on MSN5d
Scientists Solve Long-Standing DNA Machinery Mystery
SMC proteins can reverse direction, reshaping DNA and solving key scientific debates. This discovery could impact research on ...
Nature1d
NMR spectroscopy articles from across Nature Portfolio
Nuclear magnetic resonance (NMR) spectroscopy is a technique that detects the chemical environment of atomic nuclei by the absorption of radio-frequency electromagnetic radiation when in the ...