Vamorolone is approved and marketed as AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD) in the US, European Union, UK and ChinaNew ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...

Entering the age of 2 years, almost most children will start to actively explore their surroundings. However, there are some who appear lethargic due to muscle weakness in their bodies. This condition ...

Despite decades of research and the approval of several therapies to help manage symptoms, there is still no cure for Duchenne muscular dystrophy (DMD). Prevention through genetic testing, such as ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

A new drug developed by professors from the School of Pharmacy and Pharmaceutical Sciences at Binghamton University has received Food and Drug Administration (FDA) approval for the treatment of ...

There is no cure for DMD, but treatments can slow the progression of the disease and help improve quality of life. Duchenne muscular dystrophy (DMD) is a chronic condition that causes a gradual loss ...

- Potential first-in-class gene editing approach designed for dystrophin gene correction leading the body to produce a functional dystrophin protein applicable for majority of DMD patients (up to ~60% ...

The degree to which novel value elements such as insurance value impact estimated treatment value for rare, severe genetic diseases such as Duchenne muscular dystrophy is unclear. Objectives: To ...