Category Archives: News from Other Sources

MDA awarded $120,000 to Catabasis Pharmaceuticals to test two anti-inflammatory compounds, CAT-1004 and CAT-1040, in the mdx mouse model of Duchenne MD.

Funds Will Support Testing of Anti-Inflammatory Compounds in Mice

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

Twenty-four weeks of treatment with the exon-skipping drug eteplirsen resulted in significant increases in dystrophin production.

The American Academy of Neurology has released guidelines for the use of intravenous immunoglobulin, which modulates the immune system in MG, LEMS and myositis.

Researchers are studying the ‘natural history’ of centronuclear/myotubular myopathies, central core disease and multiminicore disease, with an eye to future trials.

A webinar hosted by ALS TDI reviews the biotech’s development of TDI 132 (Gilenya), a drug that modulates the immune system.

Zinc finger protein 1 (ZPR1) appears to be a ‘protective modifier’ of spinal muscular atrophy, making it a potential target in the development of therapies.

In this first of several reports on MDA’s Clinical Conference, the role of genetics and immunology in different neuromuscular diseases is described.

Repligen Corp., in Waltham, Mass., announced today that it has launched a phase 1 clinical trial designed to test a compound called RG2833 in adults with Friedreich’s ataxia (FA).