Roche updates on Elevidys gene therapy for Duchenne muscular dystrophy in EU

Elevidys is currently the only approved gene therapy that targets the root cause of Duchenne

Capricor receives FDA’s CRL for Deramiocel BLA for duchenne muscular dystrophy

Capricor’s BLA for Deramiocel received Priority Review in March 2025

Pfizer updates on Phase 3 study of investigational gene therapy for ambulatory boys with Duchenne Muscular Dystrophy

The primary endpoint in the final analysis was assessed by change in the North Star Ambulatory Assessment at one year after treatment

Sarepta refuses to comply with FDA request to stop shipping gene therapy Elevidys

FDA requests Sarepta Therapeutics to suspend distribution of Elevidys and places clinical trials on hold for multiple gene therapy products following 3 deaths

Zyuds Lifesciences receives USFDA approval for DMD treatment drug

Deflazacort is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older

India is emerging as a favourable destination to conduct clinical trials: PwC-USAIC Report

Biopharma can benefit from the critical enablers of innovation in the private healthcare system in India

NS Pharma announces FDA clearance to initiate phase II study for an Exon 44 skipping candidate

Study efficacy measures will include the expression of dystrophin protein and motor function.

Pfizer announces positive topline results from Phase 3 study of Hemophilia B gene therapy candidate

Roche to present data across neuroscience portfolio at 2022 AAN Annual Meeting

Additional data across neurological disorders, including Alzheimer’s disease, help advance the scientific understanding of these conditions and the potential impact of early treatment

Emmes acquires Casimir, its fourth major acquisition

Casimir further differentiates Emmes’ industry-leading rare disease research capabilities