FDA slaps gene therapy Elevidys with boxed warning after fatal liver failures

Elevidys came under scrutiny in June when the FDA disclosed two fatal cases in non-ambulatory boys who developed dangerously high liver enzyme levels

Novartis to acquire Avidity Biosciences for $12 billion

The acquisition will follow the planned separation of Avidity’s early-stage precision cardiology programs into a new, independent company

FDA approves Zydus’ Deflazacort oral suspension, 22.75 mg/mL

Deflazacort oral suspension is indicated for treating Duchenne Muscular Dystrophy in patients 5 years of age and older

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