National Limb Girdle Muscular Dystrophy Conference Scheduled This Fall

The National Limb Girdle Muscular Dystrophy Conference, scheduled Aug. 30 to Sept. 2 at the Hyatt Regency in Chicago, will highlight all forms of limb-girdle muscular dystrophy (LGMD) in a gathering of researchers, neurologists, biotechs, and patients. A project and program of The Speak Foundation, a nonprofit focused on improving the quality of life for . . .

Read More

DMD Patients Invited to Participate in a Survey About Symptoms and Daily Life

Researchers at the University of Rochester in New York seek patients diagnosed with Duchenne muscular dystrophy (DMD) who are interested in helping to develop disease-specific patient-reported outcome measures for future clinical trials. This study will help to determine the most critical symptoms of children, young adults, and adult patients with DMD, and as a result, . . .

Read More

Clinical Trial Alert: Alexion Pharmaceuticals Seeks Participants for a Phase 3 Pediatric Myasthenia Gravis Study

Researchers at Alexion Pharmaceuticals are looking for children with generalized myasthenia gravis (gMG) to participate in an open-label Phase 3 study. The goal of the study is to evaluate the safety and efficacy of eculizumab (Soliris) in pediatric patients. This therapy may improve muscle strength by reducing inflammation caused by the immune system. All participants . . .

Read More

Clinical Trial Alert: Biogen Seeks Participants for a Phase 3 SOD1-ALS Study

Researchers at Biogen are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in the VALOR Phase 3 study designed to help researchers evaluate the effects of BIIB067 on disease progression in patients with ALS caused by superoxide disumtase 1 (SOD1) mutation (SOD1-ALS). In ALS, motor neurons degenerate or die and stop sending messages . . .

Read More

Do You Know ALS? Meet Dr. Cudkowicz.

Dr. Merit Cudkowicz, chief of Neurology at Massachusetts General Hospital, where the ALS Multidisciplinary Clinic in the Healey Center for ALS is associated with MDA, is dedicated to performing academic-led clinical trials in amyotrophic lateral sclerosis (ALS). Her internationally renowned research has led to advanced treatments for people with living with ALS. What role has . . .

Read More

Do You Know ALS? Meet Tim.

In 2016, former NFL player Tim Green was diagnosed with amyotrophic lateral sclerosis (ALS) and was private about his new life at first. Over time, Tim has become an advocate for research to find new treatments and cures, and he found strength in his doctor and his family. He shared his story on “60 Minutes” . . .

Read More

JAMA Neurology Publishes Article on Newborn Screening and Neuromuscular Disorders

Earlier this week, JAMA Neurology published an article co-authored by Muscular Dystrophy Association (MDA) staff members that addresses the role of newborn screening (NBS) in the field of neuromuscular disease. In the article, “Maximizing the Benefit of Life-Saving Treatments for Pompe Disease, Spinal Muscular Atrophy, and Duchenne Muscular Dystrophy Through Newborn Screening: Essential Steps,” the . . .

Read More

Do You Know ALS? Meet Sunny.

Just two weeks before her 28th birthday, and after a long search for an explanation for a series of symptoms, Sunny Brous was diagnosed with amyotrophic lateral sclerosis (ALS). Sunny has worked to remain independent and raise awareness to change the future of ALS. Her mantra is, “No apologies, no excuses, no regrets.” Can you . . .

Read More