The Institute for Clinical and Economic Review (ICER) has shared the details of its future analysis comparing the clinical benefits and long-term cost-effectiveness of approved Spinraza (nusinersen) and investigational therapy AVXS-101 for people with spinal muscular atrophy (SMA). In August, the nonprofit research institute announced it would conduct a comparative analysis between Biogen‘s…
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Zosia Malkowska looks and acts like a perfectly healthy, happy 2-year-old. No one observing the girl — whose antics stole the show at the opening of last week’s 6th Polish SMA Conference in Warsaw — would guess she had been born with spinal muscular atrophy (SMA). That’s because Zosia is relatively…
A new experimental method of delivering Spinraza (nusinersen) to people with spinal muscular atrophy (SMA) could reduce both the costs and burden of the treatment, potentially allowing more patients access to the therapy, researchers say. The new approach was outlined in the study, “Preliminary Safety and Tolerability of…
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…
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…
