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

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 ...

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?

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 ...

An inherited genetic mistake prevents the ... Most children with Duchenne muscular dystrophy will need a wheelchair to get around by the time they're teenagers. The disease also damages the ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). DMD, a severe genetic disorder that causes muscle weakness and shortens lifespans, arises from mutations in ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). Published in the December issue of the Journal of Biological Chemistry, researchers characterize the ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy for Duchenne muscular dystrophy (DMD), following positive data from the ...

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 ...