Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne, commemorated its 30th ...

Milestone represents a significant step forward in the development of deramiocel for the treatment of Duchenne muscular ...

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

Regenxbio shares surged 20% after disclosing new, positive efficacy and safety data of its Duchenne muscular dystrophy treatment. Shares were recently trading around $11.55. The stock is down 46% on ...

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...

After winning an expanded approval for its gene therapy to treat Duchenne muscular dystrophy earlier this year, Sarepta ...

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...

Trump fingers Robert F. Kennedy Jr. to lead the HHS, lupus and ATTR-CM dominate headlines this week, bluebird bio has a cash gap to leap and RegenxBio eyes Sarepta in Duchenne muscular dystrophy.

Capricor Therapeutics (CAPT) stock in focus as company receives Orphan Drug and ATMP designations from the EU regulator for its lead drug deramiocel. Read more here.

Throughout 2024, the FDA issued new drug approvals and expanded treatment indications across various rheumatologic diseases.