News

Certain stem cells may offer benefits for DMD treatment: Early study

Stem cells derived from the amniotic membrane of pregnant women after childbirth “could provide therapeutic benefits” for people with Duchenne muscular dystrophy (DMD), according to researchers in Japan. These stem cells, known as human mesenchymal stromal cells, were able to delay DMD progression and preserve muscle function in a…

DYNE-101 receives orphan drug status for DM1 in Europe

Dyne Therapeutics’ investigational therapy DYNE-101 has been granted an orphan drug designation by the European Medicines Agency (EMA) for myotonic dystrophy type 1 (DM1). The designation is meant to support the development of potential therapies for rare, life-threatening, or chronically debilitating diseases. The benefits include reduced fees, clinical…

MDA creates Gene Therapy Support Network

The Muscular Dystrophy Association (MDA) has launched the Gene Therapy Support Network (GTx) that will offer resources and guidance about approved gene therapies for people living with neuromuscular diseases. Its launch comes shortly ahead of a possible U.S. approval of SRP-9001 (delandistrogene moxeparvovec), Sarepta Therapeutics‘ experimental…