For the second time, a person with DMD has died of acute liver failure after being treated with the one-time gene therapy Elevidys.

Patients are often the best experts on their disease, but what about emergency situations? Columnist Patrick Moeschen shares ...

Columnist Robin Stemple marks two anniversaries that forever altered his life. Despite the incredible challenges, he feels ...

The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an ...

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart ’s experimental stem cell ...

Duvyzat's approvals were based on the Phase 3 EPIDYS trial, which included 179 boys, ages 6 and older, who were able to walk.

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 ...

The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an investigational gene therapy for limb ...

As her only son without DMD plays in a statewide all-star football game, columnist Betty Vertin reflects on his tie to his brothers.

It’s hard to believe: This is the 100th column I’ve written for Muscular Dystrophy News Today. It will also be my last for the near future. “Hawk’s-Eye View” has been a part of me for the past three ...