Roche has reported positive topline outcomes from the second year of the multinational Phase III EMBARK trial of Elevidys ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...

It was, on the outside looking in, a marvellous Christmas for the Ennis household in Tallaght. Archie (7) was totally excited ...

An Irish woman has launched a fundraiser to help raise money for medical treatment for her seven-year-old son. Una Ennis' son ...

GIVI-MPC, a stem cell therapy for certain types of muscular dystrophy, has been granted orphan drug status for treating ...

Ips Heart Inc. has been awarded orphan drug designation by the FDA for its GIVI-MPC stem cell therapy for Becker muscular dystrophy. GIVI-MPC has the unique ability to create new muscle with full ...

According to one research review, mesenchymal stem cell (MSC) therapy may help reduce or prevent central nervous system (CNS) damage due to MS. Some participants in clinical studies also noted ...

Photograph credit Muscular Dystrophy UK. RECENTLY, the Scottish Medicines Consortium (SMC) has accepted the drug vamorolone (also sold under the brand name Agamree) as a treatment option for people ...