Duchenne muscular dystrophy (DMD) causes dysfunction of muscle cells that help move blood through the circulatory system, a ...

Life is so much easier with a sense of humor, writes columnist Patrick Moeschen, because with LGMD, there’s no shortage of ...

The FDA has granted rare pediatric disease designation to (Z)-Endoxifen as a potential treatment for Duchenne muscular ...

As 2025 draws to a close, columnist Shalom Lim looks back at the highs and lows he's experienced this year while living with ...

Dyne Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) to approve DYNE-251, its exon-skipping therapy for people with Duchenne muscular dystrophy (DMD) amenable to exon 51 ...

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.

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 ...

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.

Doctors can use several tests to diagnose muscular dystrophy (MD). A correct diagnosis gives patients an understanding of how their disease is likely to progress. It also helps doctors do a better job ...

Two years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), motor function continues to improve in boys with Duchenne muscular dystrophy (DMD) who entered a global ...

A young man with Duchenne muscular dystrophy (DMD), who was participating in Pfizer’s Phase 1B open-label clinical trial evaluating its gene therapy candidate PF-06939926, has died. The patient has ...