Neu refix granted orphan disease designation (ODD) & rare pediatric disease designation (RPDD) by US FDA for treatment of ...

Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one ...

M01, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD). The program leverages the company’s proprietary Alphastem ...

A possible gene therapy approval and a series of major trials readouts set the stage for a busy year of drug development in Duchenne muscular dystrophy.

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

The embryos were female, and Jenssen remembers the doctors assuring her that, because the Duchenne mutation is linked to the ...

A Northern Ireland man, 31, with Duchenne Muscular Dystrophy – a progressive muscle wasting disease that affects skeletal ...

Pfizer is “evaluating appropriate next steps” for its Duchenne muscular dystrophy (DMD) gene therapy after it failed a phase 3 trial. Fordadistrogene movaparvovec (PF-06939926) was unable to ...

Important insights into the mechanisms behind Fuchs Endothelial Corneal Dystrophy (FECD), a common cause of age-related visual loss, have been revealed in a new study led by UCL researchers.

Multiple sclerosis review. P T.; 37(3):175-184. Genetic and Rare Diseases Information Center (GARD). Duchenne muscular dystrophy. Genetic and Rare Diseases Information Center (GARD). Becker muscular ...