New Hope for Duchenne: Ryoncil Clinical Trial Begins
A new breakthrough may change the future of Duchenne muscular dystrophy. Ryoncil (remestemcel-L-rknd), a novel cell therapy, has received FDA clearance for a key...
Regenxbio Reports New Positive Interim Data From Phase 1/2 Affinity Duchenne Trial Of Rgx-202
REGENXBIO announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy....
Tenaya’s TN-301 Beats Givinostat in Improving DMD Muscle Function
Tenaya Therapeutics presented encouraging preclinical data evaluating TN-301, the company’s highly selective HDAC6 inhibitor, at the Muscular Dystrophy Association’s Clinical & Scientific Conference 2026...
Vamorolone (Agamree) in DMD: Real-World Experience
Santhera Pharmaceuticals announced that full results from long-term real-world comparative analyses, including baseline data from the ongoing GUARDIAN study of AGAMREE (vamorolone), were presented...
Santhera Announces Proposed Inclusion and Reimbursement of AGAMREE (Vamorolone) within Spain’s National Health System for the Treatment of Duchenne Muscular Dystrophy
Santhera Pharmaceuticals announces that the Spanish Interministerial Commission on Prices of Medicines, has proposed to include AGAMREE (vamorolone), in the pharmaceutical coverage of the...