Quest Magazine

MDA Hosts National Becker MD Conference in August

Update (Sept. 7, 2012): Videos of all presentations from the BMD Conference have been archived on MDA's BMD Conference video page. For a brief synopsis of each video, see BMD Conference Videos Cover Health Care, Research and Daily Living.

Stop Codon Read-Through Drug Performs Well in DMD Mice

An experimental drug called RTC13, designed to treat Duchenne muscular dystrophy (DMD) by restoring production of the muscle protein dystrophin, has shown promise in experiments in dystrophin-deficient mice that have a DMD-like disease.

RTC13's MDA-supported developers say they're optimistic about the compound but that refinement of its chemistry and further testing will be needed before it can be taken into clinical trials in people with DMD.

MDA Symposium Looks at Partnerships Between Industry and Academia

Enhancing collaboration and transfer of technology between academia and industry was the subject of MDA's recent Translational Research Symposium, one of a series of four MDA-sponsored research symposia scheduled in 2012.

The symposium was held June 27 in New Orleans, in conjunction with the 2012 New Directions in Biology and Disease of Skeletal Muscle Conference.

'ALS Postcards' Highlight Global Efforts to Defeat ALS

On World ALS Day, June 21, 2012, the ALS Therapy Development Institute launched "ALS Postcards" as part of an international effort to raise awareness of amyotrophic lateral sclerosis (ALS) — known as motor neurone disease, or MND, in most parts of the world.

Pioglitazone Benefits Mice But Not Man

The anti-diabetic drug pioglitazone did not improve survival time when tested as an add-on therapy to riluzole in a phase 2 clinical trial of people with amyotrophic lateral sclerosis (ALS).

The trial, which was conducted in Germany, was stopped early "for futility" when interim results showed no effects on survival. (A futility trial is one that is designed to stop at a designated point if it's clear that the drug is not having the intended effect.)

Stem Cell Briefs: Contributions of PAX7, S1P, MCAM

Update (July 10, 2012) — This story was updated to reflect the availability of a podcast with MDA research grantee Emanuela Gussoni, who discusses the development of stem-cell-based treatments for muscular dystrophies.

Patient Assistance Program Launched for Nuedexta

A new Patient Assistance Program can help people with a diagnosis of pseudobulbar affect (PBA) get a medication designed to treat the condition at low or no cost.

MDA Symposium Puts Glial Cells in the Spotlight

The contribution of nervous system support cells called glia to the degeneration of motor neurons in amyotrophic lateral sclerosis (ALS)was the topic of serious discussion among researchers at MDA's Neuron Symposium on May 22, 2012.

The meeting, held at MDA's national headquarters in Tucson, Ariz., brought together ALS researchers studying the toxic role played in that disease by glia, and scientists who specialize in these key support cells.

Podcast: Howard Worman Discusses Research Related to Heart Disease in EDMD and LGMD1B

Recently, MDA grantee Howard Worman at Columbia University, and colleagues, published encouraging results about the effects of a drug called selumetinib on the hearts of mice with a genetic mutation that's found in a form of Emery-Dreifuss muscular dystrophy (EDMD). 

MDA Urges Legislators to Speed Up Approval of Rare Disease Drugs

Update (July 10, 2012) —Reauthorization of the Prescription Drug User Fee Act (PDUFA) was signed by President Obama on July 9, 2012, complete with a provision that creates an accelerated approval pathway for drugs for rare diseases.

Pages

MDA in Your Community