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

DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

When living with Duchenne muscular dystrophy, taking a gamble feels better than not doing anything at all, columnist Betty Vertin writes.

And it's first such treatment in his class," said ... Karachunski said there are many more muscular dystrophy treatments that are currently in clinical trials that he believes could help Colton ...

Kye Pharmaceuticals, Inc. ("Kye") announced today it has submitted a New Drug Submission (NDS) to Health Canada for the ...

None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of ...

And it's first such treatment in his class," said ... Karachunski said there are many more muscular dystrophy treatments that are currently in clinical trials that he believes could help Colton ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada The Canadian regulatory submission of vamorolone ...