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

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 ...

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

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

Columnist Patrick Moeschen, anticipating his own historic anniversary, celebrates champions of the disability rights movement ...

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.