Tag: Research

Adults, News, Research, Living with Muscular Dystrophy

Clinical Trial Alert: Phase 2 Study of Ravulizumab (Ultomiris) in Adults Living with Dermatomyositis

MDA Staff

Researchers at Alexion Pharmaceuticals are seeking adults living with dermatomyositis to participate in a phase 2 clinical trial to evaluate the safety, effectiveness, and pharmacological properties of the investigational drug ravulizumab (Ultomiris). Ravulizumab is designed to target a component of the immune system (known as complement), which underlies many autoimmune disorders including dermatomyositis. Treatment with ravulizumab . . .

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Kids, Young Adults, Adults, News, Research, Living with Muscular Dystrophy

Simply Stated: What is Myotonic Dystrophy?

Sujatha Gurunathan

Myotonic dystrophy (DM) is a type of muscular dystrophy that affects about 1 in 8,000 people worldwide. The disease is known by several names, including Steinert disease, after the doctor that first described it, and “DM” in reference to its Greek name, dystrophia myotonica. Similar to other forms of muscular dystrophy, DM is characterized by . . .

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News, Research

2022 Clinical & Scientific Conference March 15 Highlights

Sujatha Gurunathan

Today was the second full day of sessions of 2022 MDA’s Clinical & Scientific Conference. The three major themes of today’s sessions were (1) therapeutic strategies to treat NMDs, (2) clinical trial design and implementation, and (3) optimizing patient care.  Highlights Mitochondrial interventions: small molecule and gene therapy Carlos Moraes, PhD, from the University of . . .

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News, Research

2022 Clinical & Scientific Conference March 14 Highlights

Sujatha Gurunathan

Today was the first full day of sessions of MDA’s 2022 Clinical & Scientific Conference. For the first time since the start of the COVID-19 pandemic, clinicians, scientists, policymakers, nonprofit, and industry leaders are convening, primarily in-person, for a dynamic and informative five days in Nashville, TN. With more than 975 in-person and 490 virtual . . .

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Kids, Young Adults, Research, Living with Muscular Dystrophy

Clinical Trial Alert: Phase 1b/2a Study of WVE-N531 in a Subset of People Living with DMD

Sujatha Gurunathan

Researchers at Wave Life Sciences are seeking boys living with DMD caused by gene mutations amenable to exon 53 skipping interventions to participate in a phase 1b/2a clinical trial. This trial will evaluate the safety, effectiveness, and correct dosage of the investigational drug WVE-N531. WVE-N531 is an exon-skipping drug designed to promote skipping over a section . . .

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Adults, Research, Living with Muscular Dystrophy

Research Study Alert: Survey of Pain Self-Management and Well-Being in People with General Pain or with Myotonic Dystrophy

Sujatha Gurunathan

Researchers at Newcastle University are seeking adults living with general pain or with myotonic dystrophy (DM) to participate in a survey to learn about the ability of individuals affected by these conditions to self-manage pain and to determine how this affects overall well-being. This study involves a 15-20 minute online survey. To be eligible, individuals must . . .

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Kids, Young Adults, Adults, Research, Living with Muscular Dystrophy

Simply Stated: What is Duchenne Muscular Dystrophy?

Sujatha Gurunathan

Duchenne muscular dystrophy (DMD) belongs to a group of rare genetic disorders, known as dystrophinopathies, that are caused by mutations of the dystrophin gene. DMD is characterized by progressive degeneration and weakness of the body’s voluntary muscles, primarily the skeletal muscles that control movement. In later stages, the heart and respiratory muscles may also be . . .

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Personal Stories, Kids, Young Adults, Adults, Research

International Day of Women and Girls in Science: MDA’s Spotlight on Dr. Angela Lek

Rebecca Hume

In recognition of International Day of Women and Girls in Science, the Muscular Dystrophy Association (MDA) is honored and excited to highlight the career and accomplishments of Dr. Angela Lek, PhD. International Women and Girls in Science Day, February 11, endeavors to acknowledge and celebrate the invaluable role that women and girls play in accelerating . . .

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News, Research

Muscular Dystrophy Association Awards 18 Grants Totaling Over $1.6 Million for Neuromuscular Disease Research

Sujatha Gurunathan

On Oct. 28, 2021 the Muscular Dystrophy Association (MDA) announced the awarding of 18 new grants totaling over $1.6 million toward neuromuscular disease (NMD) research. These new grants represent a continued commitment by MDA to fund groundbreaking research that will one day lead to treatments and cures for the diseases in its program. The newly . . .

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News, Research

Capricor Announces Positive Results from its Phase 2 Study of CAP-1002 to Treat DMD

Sujatha Gurunathan

On September 24, Capricor Therapeutics announced positive results at the World Muscle Society Virtual Congress from its phase 2 HOPE-2 trial of the investigational therapy CAP-1002 for treatment of people in advanced stages of Duchenne muscular dystrophy (DMD). The primary and secondary endpoints of the study, the improvement of upper limb and cardiac function, were met, . . .

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