Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with ...

Proton pump inhibitors are a class of medications used to treat conditions like GERD and ulcers. They work by blocking the ...

As a reminder, WVE-N531 does not leverage muscle delivery conjugates, and as such, patients are not at risk for conjugate ...

The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...

WARREN, N.J., Nov. 13, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment ...

Magnesium deficiency is also known as hypomagnesemia. This is linked to muscle cramps, fatigue, and irregular heartbeat. It may also contribute to mental health issues like anxiety. A 2020 review ...

Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...

Paroxysmal ventricular fibrillation unassociated with heart block occurred in two patients with hypomagnesemia. In neither patient were other causes of the arrhythmia apparent. Temporary transvenous ...

First-ever FDA approval for gene therapy directly administered to the brain - - Priority review voucher granted - - Broad label including children and adults - - ...

Diuretics are important therapeutic tools. First, they effectively reduce blood pressure and have been shown in numerous hypertension clinical trials to reduce both cardiovascular and ...

Expert Rev Clin Pharmacol. 2013;6(4):443-451.