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To design effective clinical trials for new therapies, researchers need to know how to measure success. Several years ago, four neuromuscular researchers realized there was a gap in the knowledge needed to develop therapies for limb-girdle muscular dystrophy (LGMD). Jeffrey Statland, MD, a professor of neurology at the University of Kansas Medical Center in Kansas…

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Next week, researchers, clinicians, industry leaders, and families will gather at the 2026 MDA Clinical & Scientific Conference, hosted by the Muscular Dystrophy Association (MDA), to discuss the latest advances in neuromuscular disease research and care. Ahead of the event, Bionews, the parent company of this site, sat down with Sharon Hesterlee, PhD, the MDA’s president…

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Researchers at NMD Pharma A/S are working to better understand generalized myasthenia gravis (gMG) and study efficacy of a potentially new treatment. The study People who have generalized myasthenia gravis (gMG) may be eligible to participate in a phase 2 clinical trial (SYNAPSE-MG) to evaluate the safety, efficacy, and duration of effect of the investigational therapy…

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Every year on Rare Disease Day, communities around the world come together to shine a light on conditions that are too often overlooked or underfunded. Historically, rare diseases have been defined by what they lack: large patient populations, widespread public awareness, and approved treatments. Yet collectively, rare diseases affect more than 30 million people in…

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I’m going on a blind date! I should mention that it is with a book. Regular readers of my column will know that I am happily married to Jason, my husband of nearly 25 years. I got the idea a couple of weeks ago after visiting an antique mall where books were being sold. They…

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Researchers at Novartis are working to better understand idiopathic inflammatory myopathies (IIMs) and to evaluate a potential new treatment for individuals living with IIM who have not responded to previous treatments (a condition known as refractory disease). The study Individuals with refractory IIM may be eligible to participate in a phase 2 clinical trial (AUTOGRAPH-IIM) evaluating…

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Researchers have identified a molecular pathway that helps regulate how muscle repairs itself — a discovery that may help guide the development of future treatments for conditions such as muscular dystrophy (MD) and severe muscle injuries, a study reports. The pathway depends on a protein called platelet-derived growth factor receptor beta, or PDGFRb, and appears…

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On Thursday, February 26, advocates, clinicians, patient leaders, and policymakers came together for a powerful Senate Special Committee on Aging hearing titled “From Regulator to Roadblock: How FDA Bureaucracy Stifles Innovation.” This conversation served as a reminder of how far the muscular dystrophy and rare disease community has come, and the work still to be…

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My family and I have never been athletically inclined. Our talents lie in other areas. That being said, we’ve enjoyed watching the recent Winter Olympics together. As a blind person, I couldn’t see the skiers, skaters, sliders, riders, jumpers, and other athletes in action, but the commentators provided descriptions that made the events accessible to…

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X-linked myotubular myopathy (XLMTM) is a rare, inherited neuromuscular condition that primarily affects infant males. It is one of the most severe forms within a group of disorders called centronuclear myopathies, which are characterized by distinctive muscle cell changes seen by biopsy and profound muscle weakness that begins early in life. It is estimated that…

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