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 ...

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

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

DMD treatment KER-065 showed a good safety profile in a Phase 1 clinical trial with no serious side effects seen in healthy ...

Betty Vertin, mother of three sons with Duchenne, shares her journey of managing treatment, mental health, and parenting ...

Hawkin Miller, who has Duchenne muscular dystrophy, shares motivation and advice for staying on track with treatment.

Shalom Lim, who has Duchenne muscular dystrophy, shares how caregivers can support patient autonomy when it comes to ...

Treatment with gene therapy candidate BB-301 led to improvements in swallowing ability for the first three OPMD patients in a clinical trial.