The FDA is allowing Sarepta to resume shipments of Elevidys (delandistrogene moxeparvovec) to ambulatory patients with Duchenne muscular dystrophy.

Since becoming Vertex's top scientist in 2015, Altshuler has helped the company become an industry powerhouse. He’ll depart ...

Bulwark reporter Will Sommer reports MAGA influencer Laura Loomer is drawing criticism from MAGA members who claim she’s ...

After Loomer targeted surgeon general nominee and Kennedy ally Casey Means, Means’s brother, Calley, suggested that Loomer’s ...

The cost of high-end, breakthrough medications traumatizes patients and their families. According to USAfacts, “national ...

Hearing minnow Audeara has entered an alliance with Ear Science Institute Australia to develop an affordable bone conduction hearing device.

Extend transfection complex formation time by up to 3 hours, reduce complex volume by >50% and maintain high titers and full ...

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

Rare disease and gene therapy stocks, battered in recent months by clinical and commercial setbacks, will likely benefit from ...

Wilson Encourages Investors Who Suffered Losses Exceeding $100,000 In Sarepta To Contact Him Directly To Discuss Their ...

I understand the heartbreak of watching someone you love spiral. But compassion cannot mean locking people up,” writes a STAT ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.