It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the importance of early diagnosis of muscular dystrophy.

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the importance of a smooth transition from pediatric to adult care for patients with ...

A recent discovery of a molecular connection between autism and myotonic dystrophy, a type of neuromuscular disease, may provide a breakthrough on how clinicians approach autism spectrum disorder.

Sarepta Therapeutics SRPT announced updates from its clinical programs, which focus on targeting different subtypes of ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Health Canada granted priority review to an application seeking approval of vamorolone, marketed in the U.S. as Agamree, for ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

Previously, Dr. Gita serves patients at the Ajiwaras Clinic, Cilandak KKO. Here are the causes and treatment for the congenital disorder Duchenne muscular dystrophy! Entering the age of 2 years, ...

Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada The Canadian regulatory submission of vamorolone ...