Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

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 . . .

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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 . . .

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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 . . .

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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” . . .

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FDA Approves AveXis’ Zolgensma for Treatment of Spinal Muscular Atrophy in Pediatric Patients

Today, the U.S. Food and Drug Administration (FDA) approved Zolgensma (onasemnogene abeparvovac-xioi), the first gene therapy for a neuromuscular disease. Zolgensma is a one-time intravenous (into the vein) infusion for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, . . .

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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 . . .

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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 . . .

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Support ALS Patients: Tell Congress to Pass the ALS Disability Insurance Act Today

Amyotrophic lateral sclerosis (ALS) is a neuromuscular degenerative disease that causes rapid muscular atrophy. It progresses quickly, and patients need access to all available support as soon as possible. Currently, all ALS patients must wait five months before they can access Social Security Disability Insurance (SSDI) benefits. Because of the fast-moving nature of this disease, . . .

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Do You Know ALS? Meet Dr. Glass.

Dr. Johnathan Glass is the director of the Emory ALS Center, co-chair of the Northeast ALS Consortium, and professor of Neurology and Pathology at Emory University in Atlanta. Dr. Glass’ laboratory focuses on the study of amyotrophic lateral sclerosis (ALS), investigating causes and potential therapies and cures. MDA has been a partner in research, providing . . .

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Do You Know ALS? Meet Jim.

In 2015, Karen Condron was honored with MDA’s prestigious Wings Over Wall Street Spirit Award in recognition of her passion for life — while fighting amyotrophic lateral sclerosis (ALS). It was a battle she fought with grace for eight years, until her passing last July. For years, Karen’s spirit —the namesake of the award — . . .

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