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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2019 Clinical & Scientific Conference Highlight: Neuromuscular Disease Impacts Cognition and Behavior, Too

Healthcare providers who work with patients diagnosed with neuromuscular disease (NMD) must consider the impact of these diseases on cognition and behavior, said presenters at a 2019 Clinical & Scientific Conference session focusing on mental health. Indeed, there is an indirect connection between NMD and mental health, but Dr. John Day from Stanford University, Dr. . . .

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2019 Clinical & Scientific Conference Highlight: Physical, Occupational, and Speech Therapy Considerations in NMD

During a late-morning session on the final day of the 2019 Clinical & Scientific Conference, five healthcare professionals — Laurie Sterling, a speech therapist at the Texas Children’s Hospital; Dr. Roxanna Bendixen, an occupational therapist and assistant professor at the University of Pittsburgh; Jennifer Chapin, a speech therapist at the University of Florida; Dr. Melissa . . .

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The Christopher Project Report to the Myotonic Dystrophy Community is Now Available

The results of the Christopher Project have been published in a comprehensive, 80-page report. The creation of “The Christopher Project Report to the Myotonic Dystrophy Community” was supported by several organizations, including the Muscular Dystrophy Association (MDA), and is available for download on MDA’s website. (Printed copies are also available upon request.) What is the . . .

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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 . . .

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MDA Awards Venture Philanthropy Funding to Locana to Develop Novel Treatment for DM

Today, MDA and Locana, a leading RNA-targeting gene therapy company, announced the award of an MDA Venture Philanthropy (MVP) grant totaling $550,000 to advance Locana’s development program for myotonic dystrophy (DM), the most common form of adult-onset muscular dystrophy. MVP is the MDA’s drug development program that is exclusively focused on funding the discovery and . . .

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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, . . .

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