A Phase 2 clinical trial is testing the effectiveness of Mestinon (pyridostigmine) — an approved medicine for another neuromuscular disease — in treating fatigue in people with spinal muscular atrophy (SMA) types 2, 3 and 4. The trial’s…
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A noninvasive method of detecting the presence of the SMN protein in blood samples may become the gold standard for evaluating and monitoring new therapeutic strategies for spinal muscular atrophy (SMA), new research indicates. The study, “A new biomarker candidate for spinal muscular atrophy: Identification of a peripheral…
Researchers have identified outcome measures and biomarkers of disease progression that may allow them to differentiate between types of spinal muscular atrophy (SMA) and could be beneficial in evaluating current and future therapeutic approaches. Their findings were published in the study, “Prospective and longitudinal natural history…
Spinal muscular atrophy (SMA) is just as serious a problem in Poland and other former Soviet satellite states and republics, yet access to the latest in therapies and information about the disease still lags behind that of North America and Western Europe. To address those shortcomings, SMA Foundation…
Psychological interventions to control emotional stress can help relieve the anxiety that parents and children with spinal muscular atrophy (SMA) type 1 feel during Spinraza administration through spinal taps, an Italian study reports. Researchers recommend that similar interventions become part of the…
Devastating. Heartbreaking. A death sentence for an estimated 1,000 children with spinal muscular atrophy (SMA). These are among the many angry reactions to Tuesday’s appraisal consultation document issued by a committee of Britain’s independent, government-funded National Institute of Health and Care Excellence (NICE). That opinion recommends against Spinraza (nusinersen), an…
The National Institute for Health and Care Excellence (NICE), the U.K. agency that decides on the cost-effectiveness of therapies for inclusion in the public health system of England and Wales, is recommending against Spinraza (nusinersen) being offered to treat people with spinal muscular atrophy (SMA). This stance, released as “draft…
At sunset on the second Saturday of every August, it’s a tradition to light candles in memory of those who have died of spinal muscular atrophy — and to honor those currently living with the disease. Cure SMA says the candle-lighting ritual — which this year falls on Saturday, Aug. 11 — is only one way to spread the word during SMA Awareness Month. Others include attending an Awareness Day with a local major or minor league baseball team, requesting a proclamation from state or local government, hosting an awareness-themed fundraiser, contacting local media or celebrities, and participating in social media awareness efforts. Upcoming baseball games with an SMA Awareness Month theme will be sponsored Aug. 19 by the Chicago White Sox at Guaranteed Rate Field; Aug. 20 by the Boston Red Sox at Fenway Park; Aug. 24 by the Arizona Diamondbacks at Chase Field in Phoenix; Aug. 26 by the New Hampshire Fisher Cats in Manchester; and Aug. 26 by the Tampa Bay Rays at St. Petersburg’s Tropicana Field. SMA, the leading genetic cause of death for infants, affects the motor nerve cells in the spinal cord, robbing patients of the ability to walk, eat or breathe. About one in 11,000 babies is born with SMA; nearly eight million Americans are genetic carriers. Cure SMA had a major breakthrough in December 2016, when the U.S. Food and Drug Administration (FDA) approved Spinraza (nusinersen), manufactured by Biogen. The FDA approved Spinraza without restrictions, and insurance generally covers 90 percent of cases for types 1, 2 and 3. Cure SMA now represents about 7,000 of the nation’s estimated 10,000 SMA patients, according to a recent speech by its president and CEO, Kenneth Hobby. The Chicago-based nonprofit — founded in 1984 — operates on an annual budget of $11 million. Hobby said it has spent $70 million to date on research, and some 4,000 families benefit from its services, Each year, the organization asks supporters to submit their SMA Awareness Month photos to its Facebook page. The group can also be tagged on Twitter and Instagram using the #cureSMA hashtag. Alternatively, photos may be emailed to [email protected] with “SMA community album” in the subject line. For more information, visit Cure SMA’s events calendar, download an awareness and advocacy kit (including key information about SMA that can be useful when composing a blog post or sponsor request letter), or request a medical professional awareness kit to share with primary care providers, neurologists, nurses or therapists. In Australia, where the government recently authorized insurance coverage for Spinraza for people under 18, Melbourne-based SMA Australia will host its inaugural SMA 24-Hour Challenge on Aug. 30. The organization supports people living with SMA through its cough assist and well-being programs, as well as through palliative care. “Our federal government has done an incredible job to make the drug accessible to children, but more needs to be done for adults,” said SMA Australia CEO Juli Cini, who lost two children to the disease. “I encourage everyone to get behind our 24-Hour Challenge, which will further help us in our work that supports Australians living with SMA and their families.”
The Institute for Clinical and Economic Review (ICER) will compare the clinical effectiveness and value of the only approved disease-modifying treatment for spinal muscular atrophy (SMA) — Spinraza (nusinersen, by Biogen) — and an investigational gene therapy showing promise in advanced clinical trials — Novartis‘ AVXS-101,…
Researchers at Mizzou — the University of Missouri — have started a new company, Shift Pharmaceuticals, to test the clinical effectiveness of a molecule that may help improve the outcomes of people with spinal muscular atrophy (SMA). SMA develops due to mutations in the survival motor neuron-1…
