Quest Magazine

Turning Myotonic Dystrophy On and Off in Cells

MDA grantee Matthew Disney is a current and former MDA grantee at the Scripps Research Institute in Jupiter, Fla. Disney's current MDA grant is focused on targeting toxic RNA in type 2 myotonic dystrophy (MMD2, or DM2).

Computer Error Results in Wrong Participants Getting Placebo in Tirasemtiv Trial

South San Francisco biotechnology company Cytokinetics announced July 8, 2013, that a computer program error occurred in its phase 2b BENEFIT-ALS clinical trial designed to evaluate the safety, tolerability and potential efficacy of tirasemtiv in people with amyotrophic lateral sclerosis (ALS)

DMD/BMD: Questions About Ataluren’s Mechanism

It's been widely accepted that the mechanism by which the experimental drug ataluren appears to benefit walking ability in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is that it causes "read-through" of premature stop codons — genetic instructions that cause cells to stop making a protein before the process is complete.

DMD: Drisapersen Receives ‘Breakthrough Therapy’ Designation

Multinational pharmaceutical company GlaxoSmithKline (GSK) has announced that its experimental Duchenne muscular dystrophy (DMD) drug drisapersen has received breakthrough therapy designation from the U.S. Food and Drug Administration.

A ‘Transport Problem’ May Cause Motor Neuron Death

Axonal transport is the process that is responsible for moving nutrients, proteins and other vital cellular cargo to and from the cell bodies in motor neurons – the muscle-controlling nerve cells that are lost in amyotrophic lateral sclerosis (ALS).

New Muscular Dystrophy Drugs Offer Hope

26th Annual MDA Ride for Life Breaks $1 Million Mark to Help Fight Muscle Disease

DMD UPDATE: Drisapersen Outperforms Placebo in Walking Test

Lowe's Raises $8.8 Million for MDA's Shamrocks Campaign

MDA Scientific Conference Blog: Playing in the Same Sandbox

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