Research

Progress Now Summer 2016

Results from an MDA-supported multicenter phase 1b clinical trial have shown that treatment with pyrimethamine was safe and well-tolerated and associated with reduced levels of SOD1 protein in people with ALS caused by a mutation in the gene for SOD1.Pyrimethamine is a small molecule approved by the U.S. Food and Drug Administration for treatment in humans of the parasitic infections malaria and toxoplasmosis.

Searching for Answers Across Diseases

The $11 million commitment, MDA’s largest such award in three years, underlines MDA’s dedication to investing in research across diseases with the expectation that increased understanding in one disease will inform progress and lead to research and medical breakthroughs in other disease spaces.

Progress Now Spring 2016

Researchers are looking for people with ALS to participate in a phase 1 clinical trial, sponsored by Biogen and Ionis Pharmaceuticals, to test the experimental drug Ionis-SOD1Rx. Ionis-SOD1Rx is designed to block production of flawed SOD1 protein in people with ALS caused by defects in the SOD1 gene. It is administered by intrathecal injection, in which a small needle is inserted into a space in the lower back below the end of the spinal cord in a medical procedure commonly referred to as a “lumbar puncture.”

Progress Now Winter 2016

At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently needed answers to our families. Take a look at what's happening in research and clinical trials for Winter 2016.

Progress Now Fall 2015

As part of an effort to refocus its research grants program, MDA began funding 36 new research and development grants, totaling nearly $10 million, on Aug. 1, 2015. The awards were made to researchers seeking to increase understanding of and find promising treatments and cures for muscular dystrophy, ALS and related neuromuscular diseases that limit strength and mobility.

Follow the Biomarkers

Imagine being in a strange city. You’re driving to the airport to catch a flight. Now suppose every street sign and traffic light you see is covered in paint. Which way do you turn? You look at your dashboard. All the indicators are covered in paint, too. Is the tank full or empty? Is the engine hot? Should you go faster? Slower? Or stop altogether?

In Focus: Limb-Girdle Muscular Dystrophy

Say “limb-girdle muscular dystrophy” to most people, and you’ll get a questioning look. They may wonder what kind of girdle is being talked about and how limbs fit into the picture.The name actually comes from an anatomical term describing the supporting structures of the shoulders and hips, including bones and muscles. Limb-girdle muscular dystrophy (LGMD) is a genetic disorder in which the muscles surrounding the hips and shoulders degenerate.

SMA: Full Speed Ahead

August is often referred to as Spinal Muscular Atrophy (SMA) Awareness Month, but for MDA, work on finding treatments for this degenerative disease — and providing assistance to individuals and families affected by it — is a year-round event.Today, research aimed at slowing, stopping or even preventing spinal muscular atrophy (SMA) is advancing with more speed and greater momentum than ever before. Here’s a roundup of some of the exciting research MDA has supported in the fight against SMA.

CMD: Aiming Simultaneously at Two Biological Targets

Researchers at Boston University, supported in part by MDA, say their experimental two-pronged strategy for merosin-deficient congenital muscular dystrophy (MDC1A) was highly successful in a mouse model of this disease and should be further investigated as a potential treatment approach for patients.

UPDATE: ACE-031 Clinical Trials in Duchenne MD

Update (May 2, 2013): In a May 2, 2013, press release, Acceleron Pharma and Shire PLC announced that they have concluded their collaboration on ACE-031 and related molecules, and will not restart the development of this program. John Knopf, CEO of Acceleron, said, "While we are disappointed in the outcome of the ACE-031 program, the research provided valuable insights that can be applied across Acceleron's pipeline of novel compounds and may benefit other patients in the future."

MDA Resource Center: We’re Here For You

Our trained specialists are here to provide one-on-one support for every part of your journey. Send a message below or call us at 1-833-ASK-MDA1 (1-833-275-6321). If you live outside the U.S., we may be able to connect you to muscular dystrophy groups in your area, but MDA programs are only available in the U.S.

Request Information

Our Story

Our Team

Resources

Care Centers

Community Education

MDA Resource Hub

Summer Camp

Our Approach

For Researchers

For Clinicians

Participate

Donate

Partner