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 ...

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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 ...