The FDA cleared Sarepta to start dosing in a first-in-human clinical trial testing the gene therapy SRP-9005 in people with ...

After a primary care visit, columnist Robin Stemple faces a battery of tests and appointments, which he calls a medical merry ...

Avidity's delpacibart etedesiran (del-desiran) was granted orphan drug status in Japan as a treatment for myotonic dystrophy ...

Learning life skills to handle Duchenne muscular dystrophy can include health management, working with caregivers, and ...

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

I recently had the opportunity to attend a Singaporean theater production titled “Supervision,” thanks to the SingHealth Patient Advocacy Network (SPAN). SPAN is a collective of patients and ...

The determination comes after recruitment and dosing in certain trials were paused after a young patient died due to acute liver failure.

This August will mark 40 years since I was diagnosed with what we eventually discovered was limb-girdle muscular dystrophy. Though it’s taken me many years to accept my condition, it’s progressed ...