Participants Needed for a Pilot Study of a New Diagnostic Device

Dr. Perry Mansfield at the SENTA Clinic in San Diego, Calif., is looking for healthy individuals and patients with neuromuscular disease who have documented neurologic pharyngeal muscle dysfunction (dysfunction of the voluntary muscles that form the pharynx) to participate in a pilot study. This study will help to prove the diagnostic consistency of transmembrane electromyography . . .

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MDA Awards 25 Grants Totaling More Than $6.6 Million for Neuromuscular Disease Research

MDA announced today the awarding of 25 new MDA grants totaling more than $6.6 million toward research focused on a variety of rare neuromuscular diseases (NMDs), including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), congenital muscular dystrophy (CMD), Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), Friedreich’s ataxia (FA), inclusion body myositis (IBM), limb-girdle muscular dystrophy . . .

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Biogen Announces Positive Long-Term Results from Phase 2 Study of Spinraza to Treat SMA in Pre-Symptomatic Infants

Biogen announced positive long-term results from its ongoing phase 2 (NURTURE) clinical trial evaluating Spinraza (nusinersen) for treating spinal muscular atrophy (SMA) at the Cure SMA Annual Conference held June 28 through July 1 this year. New data from the NURTURE study demonstrated that after almost four years, infants who were treated with Spinraza before developing symptoms of SMA . . .

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The ALS Association, ALS Finding A Cure, and MDA Co-Fund Clinical Trial Grant to Study Restoring Immune System Function to Treat ALS

The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The principal investigator is Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of . . .

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Scholar Rock Announces Positive Results from Phase 1 Study of SRK-015 to Treat SMA

Scholar Rock Holding Corporation announced positive results from its phase 1 clinical trial evaluating SRK-015, a therapy for treating spinal muscular atrophy (SMA), at the Cure SMA Annual Conference held June 28 through July 1 this year. Consistent with previously announced interim findings in February, the final results showed positive safety, pharmacodynamic, and pharmacokinetic data in healthy volunteers, supporting . . .

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US House Passes Newborn Screening Reauthorization

Thanks in large part to the work of MDA advocates across the country, the US House unanimously moved yesterday to pass the Newborn Screening Saves Lives Reauthorization Act (HR 2507). Once enacted, this law will reauthorize the national newborn screening program for five years and make additional investment in order to modernize and make improvements . . .

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MDA Engage Community Education Seminars Coming to an Area Near You

We believe in the power of community and the importance of building relationships with families going through similar experiences. If you are an individual living with a neuromuscular disease, we invite you and your loved ones to attend one of our MDA Engage education events taking place across the country. MDA Engage Community Education Seminars . . .

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MDA Awards Venture Philanthropy Funding to Rachelle Crosbie to Develop a Novel Membrane-Stabilizing Drug for DMD

MDA has awarded MDA Venture Philanthropy (MVP) funding totaling $389,463 over two years to Rachelle H. Crosbie, PhD, professor and chair of Integrative Biology and Physiology at the University of California, Los Angeles. The award will support the development of a small molecule drug that increases expression of sarcospan, a protein that may help to . . .

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2109 Clinical & Scientific Conference Highlight: Healthcare Technology Moves Out of the Clinic, Into the Pocket

Computers, laptops, tablets, and smartphones have become ubiquitous features of our daily lives — but not of our healthcare. At least, not yet. But a group of physicians at MDA’s 2019 Clinical & Scientific Conference made a few convincing cases for increased use of personal electronics in healthcare and clinical trials. Dr. James Berry of . . .

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2019 Clinical & Scientific Conference Highlight: What’s New in Neuromuscular Diseases?

At the 2019 MDA Clinical & Scientific Conference, one scientific session of interest centered on what’s new in neuromuscular diseases (NMDs). In this session, presenting scientists discussed progress in therapy development and clinical trials for a range of NMDs, including Becker muscular dystrophy (BMD), congenital muscular dystrophy (CMD), congenital myasthenic syndromes (CMS), myotonic dystrophy (DM), . . .

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