Scotland’s NHS can now offer givinostat to ambulant children with Duchenne muscular dystrophy, marking a step forward in care ...

Average Upside Potential as of December 10: 104.08% ...

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people.

Once the exchange agreements close, Sarepta will have $893.4 million in debt due in 2030 following previous refinancing it announced in August.

Wondering if Sarepta Therapeutics is a beaten down biotech bargain or a value trap? This article will walk through the numbers in plain English so you can decide with confidence. Despite a brutal long ...

The firm plans to seek accelerated approval from the FDA next year, while it launches a Phase III trial to support other regulatory applications.

Wedbush upgrades Sarepta Therapeutics, citing overdone selloff and potential upside as FDA could re-approval Elevidys for non ...

This article has been revised and clarified. Pasadena-based biopharma company Arrowhead Pharmaceuticals Inc. had a ...