Category Archives: News from Other Sources

Neuralstem is seeking permission to evaluate efficacy in its phase 1 safety trial, as well as to increase the dosage and number of trial participants

Mutations in the DYNC1H1 gene cause a rare form of spinal muscular atrophy; plastin 3 contributes to movement problems in SMN-related SMA

Adults with MMD1 and minor cardiac conduction defects benefit from invasive testing and possible pacemaker insertion.

A study of 22 couples considering preimplantation genetic diagnosis (PGD) has revealed a four-phase decision-making process

Findings about ANO5 mutations, DNAJB6 mutations and dysferlin gene transfer were presented

The Muscular Dystrophy Association announced today that an MDA-supported research team in Italy and Japan has identified a promising new target at which to aim experimental therapies to treat facioscapulohumeral muscular dystrophy (FSHD).

Researchers have identified a molecular switch that appears to lead to harmful gene activation in facioscapulohumeral MD.

Recent developments in Charcot-Marie-Tooth disease include CMT2E research mice, CMT1A stem cells, progress toward CMT1A gene therapy and a new pediatric disability scale.

Encouraging findings about eculizumab and GM-CSF were presented

MDA has awarded $1.5 million to ReveraGen BioPharma to develop a drug with the benefits of prednisone, but without the side effects, for Duchenne MD.