Nxera secures exclusive APAC rights to DMD drug Vamorolone in deal with Santhera

Nxera will take full responsibility for regulatory approvals, clinical development where required, manufacturing and commercialization in the licensed territories

Italfarmaco and JCR Pharmaceuticals partner to bring Duchenne therapy to Japan

The partnership also sets the stage for broader collaboration on rare disease treatments

Novartis scores FDA nod for game-changing SMA treatment

Itvisma is a one-time, fixed-dose therapy designed to tackle the genetic root cause of SMA

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

DINAMIQS inaugurates new cGMP viral vector manufacturing facility in Switzerland

The new site enables end-to-end production of viral vector gene therapies

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

Teijin Pharma receives approval for additional indication for XEOMIN in Japan

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