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 ...

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

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 ...

An independent data monitoring committee (DMC) favors continuing dosing Elevidys (delandistrogene moxeparvovec-rokl) to people with Duchenne muscular dystrophy (DMD) in ongoing clinical trials, ...