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

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

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.

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

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

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

Betty Vertin is a mother and writer living in rural Hastings, Nebraska, with her husband and seven children. Betty is a caregiver to three sons, Max, Rowen, and Charlie. Her oldest was diagnosed with ...

Muscular dystrophy refers to a group of genetic diseases that cause progressive muscle weakness and loss. There are more than 30 types of muscular dystrophy, all with different causes and symptoms.

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.

Ullrich congenital muscular dystrophy (UCMD) is a rare hereditary muscle condition that manifests at birth or a few months after birth. It belongs to a group of disorders called collagen type ...

Exon skipping is a treatment approach for people whose Duchenne muscular dystrophy is due to certain mutations in its causative gene. Exon skipping works like a molecular patch, so that the DMD gene ...