Quest Magazine

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).

MDA Offers Free Flu Shots to Those Affected by Muscle Disease

ALS Briefs: Diet, Autoimmune Disease, ALS TDI, Neuralstem, Cytokinetics

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

2013 BMD Conference Covered Research, Medical Care and Daily Life

The 2013 MDA-supported Becker Muscular Dystrophy Conference, held Aug. 24 in Baltimore and co-hosted by MDA and Johns Hopkins Medicine, was "high-energy" with "phenomenal" presenters, says Annie Kennedy, MDA's senior vice president of advocacy, who attended and helped organize the meeting.

Some 130 people attended in person and many more, including some from outside the United States, attended online.

Fairfax County Fire Fighters Hold Record-Breaking Fill the Boot for MDA

Companies to Develop Antisense Drug for Myotonic Dystrophy

 Biotechnology companies Isis Pharmaceuticals and Biogen Idec have entered into a multi-year collaboration to advance antisense technology for the treatment of neurological diseases — including type 1 myotonic dystrophy (MMD1, or DM1).

ALS Briefs: Protein Clumps, Cell-to-Cell Spread and More

This roundup of recent MDA-supported amyotrophic lateral sclerosis (ALS) research news and reviews includes:

UPDATE: Pfizer and Repligen to Collaborate on SMA Therapy Development

The biotechnology firm Repligen today announced it has entered into an agreement with the global pharmaceutical company Pfizer to advance Repligen’s spinal muscular atrophy (SMA) program, which includes RG3039, an experimental drug for SMA whose development MDA has supported.

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