What’s Next for Patients with Duchenne Muscular Dystrophy (DMD) After Pharma Giants Pull Back from AAV-Based Gene Therapy Research?
Adeno-associated virus (AAV) vectors were once heralded as the answer to delivering gene therapies, but in recent years, their prominence has diminished. It was...
Del-zota (EXPLORE44) Provides Nearly 30% Normal Dystrophin Production in Patients Amenable to Exon 44 Skipping
A phase 1/2 EXPLORE44 study (NCT05670730) is now being conducted to evaluate Del-zota, which was developed with the intention of restoring the dystrophin reading...
Dr. Aravindhan Veerapandiyan, a neurologist at Arkansas Children’s Hospital, Reviews RGX-202 Gene Therapy Study
At the American Academy of Neurology Annual Meeting, which took place in San Diego from April 5–9, 2025, the study findings of RegenxBio's RGX-202...
Givinostat EMA Approval: Is Givinostat (Duvyzat) Approved by EMA?
Duvyzat (Givinostat) has received regulatory approval in the US and UK and has completed its evaluation by the European Medicines Agency (EMA). EMA has...
Satellos SAT-3247 Stem Cell Approach May Restore Muscle Regeneration in Duchenne Muscular Dystrophy
Genetic interventions such as exon skipping or gene therapy aim to compensate for the loss of dystrophin, but do not replace the missing signal...