Quest Magazine

LEMS: Jacobus Expands Trial of 3,4-DAP

Jacobus Pharmaceutical of Princeton, N.J., is continuing its placebo-controlled trial of 3,4-diaminopyridine (3,4-DAP) for people with Lambert-Eaton myasthenic syndrome (LEMS) who have been receiving the drug through Jacbobus' "expanded access" (also called "compassionate use") program. The study, which began in January 2012, was originally by invitation only.

Prosensa Says It Remains Committed to DMD

"Our commitment to Duchenne muscular dystrophy remains," said Hans Schikan, CEO of Netherlands-based biotechnology company Prosensa at a Sept. 27, 2013, presentation in New York.

MDA's Board Chairman Advocates Support for Newborn Screening

UPDATE: DMD: Eteplirsen Shows Continued Benefit at 84 Weeks

Eighth Annual Tradition Of Hope Gala To Support MDA's Augie's Quest

ALS Briefs: Neuralstem, Body Fat, Behavior, Respiratory Care

This roundup of recent amyotrophic lateral sclerosis (ALS) research and clinical trials news includes:

Titin Mutations Can Cause CNM

Researchers in the United States and France, supported in part by MDA, have established that mutations in the titin gene are a cause of centronuclear myopathy (CNM), a group of muscle disorders characterized by variable degrees of weakness and cell nuclei that are abnormally located toward the center of muscle fibers rather than around the perimeter.

DMD: Experimental Exon-Skipping Drug Drisapersen Disappoints

A large-scale, phase 3 trial of the experimental exon-skipping drug drisapersen, in development to treat Duchenne muscular dystrophy (DMD), found no statistically significant differences on tests of walking distance or motor function between trial participants treated with the drug and those treated with a placebo.

Mouse Model of Adult-Onset SMA Encourages Researchers

'Gentler' Corticosteroid Drug Shows Promise in DMD Mice

A drug that may provide the benefits of corticosteroid medications such as prednisone and deflazacort without some of their notorious side effects — growth retardation, bone loss and suppression of the immune system — has been shown to protect and strengthen muscles in mice with a disease that mimics Duchenne muscular dystrophy (DMD).

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