The Cure SMA educational campaign is designed to equip parents and healthcare professionals with the tools needed to recognize motor delays in spinal muscular atrophy (SMA) patients early, allowing infants and children to receive potentially life-saving treatment. Called SMArt Moves, the program cites the fact that Spinraza and…
Pharmacologist C. Frank Bennett, PhD, and biochemist Adrian R. Krainer, PhD, have been awarded a prestigious 2019 Breakthrough Prize in Life Sciences for their role in the development of Spinraza (nusinersen), a therapy for spinal muscular atrophy (SMA). Bennett, of Ionis Pharmaceuticals, and Krainer, of Cold Spring Harbor…
Aiming to establish a standard of care for people affected by spinal muscular atrophy (SMA), Cure SMA has unveiled what’s being touted as the first national clinical data registry specific to this neuromuscular disease. Cure SMA will invest $9.1 million over three years in the registry, part of a…
A $123,933 grant will boost the efforts of University of Oxford scientist Carlo Rinaldi, MD, PhD, to advance understanding of spinal and bulbar muscular dystrophy (SBMA) in the hopes that it will eventually lead to a treatment. The three-year grant is being co-funded by the…
As a youth, Charles Deguire did everything he could to help three uncles with muscular dystrophy. One of them, Jacques Forest, had designed a clever makeshift arm that could pick up objects. He built it with whatever was available — including windshield wiper motors, parts of a desk lamp, and…
Kavita Krishnaswamy is so determined to gain independence for herself and others living with physical disabilities that she spends up to 20 hours a day designing devices. “After I get ready for caregivers and have something to eat, I start working, working, working,” she said in an interview with SMA…
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