Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early diagnosis, multidisciplinary care, and emerging treatments like gene therapy ...

An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines ...

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

A man with a muscle-wasting disease is pleased Manitoba is asking a federal agency to reconsider its opposition to a treatment for adults over 25, but he argues that time is running out in his case.

Spinraza remains a big product for Biogen, but has seen its sales go into reverse as competition in SMA treatment has emerged ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...

Regulators in the U.S. and the European Union agreed to review Biogen’s applications for approval of a higher dose Spinraza ...

Molgat YM, Rodrigue D . Correction of blepharoptosis in oculopharyngeal muscular dystrophy: review of 91 cases. Can J Ophthalmol 1993;28:11–4.

Applications are based on data from the DEVOTE study, which demonstrate the potential for the investigational higher dose regimen of nusinersen to advance the treatment of SMACAMBRIDGE, Mass., Jan. 23 ...