Novartis delpacibart braxlosiran (del-brax) Phase I/II study in facioscapulohumeral muscular dystrophy (FSHD) meets primary biomarker endpoint

Ad hoc announcement pursuant to Art. 53 LR FORTITUDE study met primary and key secondary endpoints of reduction in KHDC1L and ...
Medical Xpress

What you should know about spinal muscular atrophy

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...
The American Journal of Managed Care

Looking to the Future in MD Treatment

Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...
News Medical

IU study sheds new light on the development and treatment of rare form of muscular dystrophy

A study by Indiana University School of Medicine researchers sheds new light on the development and treatment of a rare form of muscular dystrophy. The study's findings were recently published in ...
Healio

Zolgensma improves motor function at 52 weeks in spinal muscular atrophy

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Motor function and other motor milestones were ...
MSN on MSN

Novartis reports positive data for neuromuscular disorder drug

Novartis NVS announced positive data from the biomarker cohort of the FORTITUDE phase I/II study. The FORTITUDE phase I/II ...