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

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

DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...

Kye Pharmaceuticals, Inc. ("Kye") announced today it has submitted a New Drug Submission (NDS) to Health Canada for the regulatory review and approval of AGAMREE® (vamorolone). If approved, AGAMREE® ...

Michelle Werner received her son’s Duchenne muscular dystrophy diagnosis with fear and desperation. Now, as CEO of Alltrna, she’s finding better ways to develop treatments.

Global Duchenne Muscular Dystrophy Drugs Market value attained USD 3.9 Billion in 2025. The market will achieve USD 6.5 ...

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

Suneel Ram's caregivers share some of the lessons they have learned while caring for the 28-year-old who was diagnosed with ...

National Policy for Rare Diseases 2021 addresses diagnosis and treatment, categorizing diseases and advocating for affordable ...

Suneel Ram is a spiritual seeker, sings in a band and was the subject of a comic novel two years ago about living with Duchenne muscular dystrophy, a rare genetic ...

Patients in Scotland can't get the treatment - which is available in England - despite manufacturers giving it away for free ...