Epicrispr Biotechnologies is partnering with Forge Biologics to support the development of EPI-321, an one-time epigentic ...

Regulators in the European Union granted orphan drug designation to SGT-003, an experimental gene therapy for Duchenne ...

As columnist Shalom Lim continues to process the loss of a dear friend, he explains why DMD advocacy work is his calling in ...

Columnist Betty Vertin shares a conversation with her daughter Lexi about finding out she was not a DMD gene carrier.

The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree (vamorolone) in the ...

Columnist Betty Vertin saw her oldest sons lose their ability to move, but that doesn't make seeing it happen to her youngest son any easier.

Due to extremely limited mobility, columnist Robin Stemple has already fallen twice in recent weeks. The next time could be ...

CureDuchenne hosts its Futures National Conference in May, with sessions on research, care, and support for Duchenne and ...

Going to school for the first time is an important moment in a child’s life. The educational journey for a child with Duchenne muscular dystrophy (DMD) can be exciting but also challenging. To smooth ...

Histone deacetylase inhibitors are a treatment approach designed to work by blocking an enzyme that turns off gene activity. This may ultimately help by slowing muscle loss and disease progression.

A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the ...

While Duchenne muscular dystrophy (DMD) is one of the most common types of this group of muscle-wasting disorders, affecting about 1 in 3,500 male births, each patient’s journey with this form of ...