Duchenne muscular dystrophy (DMD) is a genetic condition ... where one or more exons are not present. People inherit DMD in an X-linked recessive pattern. This means the gene variation is present ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Duchenne muscular dystrophy occurs when that gene fails ... Most of the muscular dystrophies are a form of inherited disease called X-linked disorders or genetic diseases that mothers can transmit ...

Prenatal diagnostic testing can now determine whether a fetus carries a debilitating or fatal sex-linked mutation. But with such screening, why hasn't the disease allele frequency gone down ...

Duchenne muscular dystrophy (DMD) is perhaps the best known of the congenital muscular dystrophies. It is an X-linked disorder that is invariably progressive and fatal, affecting 1 in 3500 newborn ...

What Is Duchenne Muscular Dystrophy? Muscular dystrophies are a group of diseases that make muscles weaker and less flexible over time. Duchenne muscular dystrophy (DMD) is the most common type.

Duchenne Muscular Dystrophy (DMD) is a rare progressive disorder. People with DMD are missing muscle-protecting protein. This is due to a fault in the gene producing dystrophin. Muscles to become ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy, did not achieve its primary endpoint, according to findings ...

A clinical trial of a gene therapy for Duchenne muscular dystrophy has been halted after a patient suffered serious side effects following treatment, Reuters reports today (November 12). After ...

Sarepta Therapeutics has decided not to continue the development of vesleteplirsen, a follow-up to its Duchenne muscular dystrophy (DMD) therapy Exondys 51. The drug – formerly known as SRP-5051 ...

Santhera Receives Positive Recommendation from NICE for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy AGAMREE® has been recommended by NICE for treating Duchenne muscular ...