Quest Magazine

Study Probes Impact of Early-Onset MMD1

Communication difficulties, social role limitations, problems with mobility and walking, and cognitive impairment were the most frequently mentioned themes in open-ended interviews conducted with people affected by congenital-onset or childhood-onset type 1 myotonic muscular dystrophy (MMD1, or DM1) or their parents.

Darius Rucker To Perform Hit Single on MDA Show of Strength Telethon Labor Day Weekend

IBM: Novartis Drug Gets Breakthrough Therapy Designation from FDA

Multinational pharmaceutical company Novartis has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for BYM338, an experimental drug it is developing to treat sporadic inclusion-body myositis (IBM).

UPDATE: FDA Approves Testing Multiple Sclerosis Drug in ALS

The U.S. Food and Drug Administration (FDA) has given the go-ahead to the nonprofit biotech ALS Therapy Development Institute (ALS TDI) to conduct a clinical trial of TDI132  — also known as fingolimod, or the brand name Gilenya— in people withALS (amyotrophic lateral sclerosis).

Live Stream of MDCC Meeting Shows Advocacy in Action

Want to spend Monday watching advocacy in action advancing the cause of muscular dystrophy research?

Becker MD Conference Can Be Attended Online Aug. 24

MDA and John Hopkins Medicine will present the 2013 national Becker Muscular Dystrophy Conference Saturday, August 24, in Baltimore. Free to all participants with BMD and their family and friends, the conference can be attended in person or online.

MDA's 'Bridge-to-Industry' Program Awards Second Grant

Christopher Penton recently completed his doctorate (Ph.D.) in integrated biomedical sciences at Ohio State University in Columbus.

MDA Awards $8.5 Million to 31 Neuromuscular Disease Research Projects

In its summer 2013 round of research grant awards, the Muscular Dystrophy Association aims to catalyze research progress in a dozen neuromuscular diseases, with an eye toward applying that knowledge to related muscle diseases, as well.

“A large number of our grants are investigating new therapeutic technologies,” notes Jane Larkindale, MDA's vice president of research. “These are 'platform' technologies, where successes can be transferred well beyond the specific disease in which they are developed and tested.”

New MDA Grants Examine Protein Misfolding in ALS

MDA’s latest round of ALS research grants focuses on basic mechanisms in the disease process, with an emphasis on targeting misfolded proteins such as TDP43, FUS and SOD1.

DMD: Exon-Skipping Drug Drisapersen Appears to Increase Dystrophin Levels

Multinational pharmaceutical company GlaxoSmithKline (GSK) has released encouraging results about dystrophin protein production in a phase 2, non-U.S. trial of its exon-skipping compound drisapersen in boys with Duchenne muscular dystrophy (DMD).

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