Poland is rolling out Biogen’s spinal muscular atrophy (SMA) therapy Spinraza (nusinersen) at a faster pace than any country that has approved it, Polish experts say. Since the first injection in May 2019, about 400 of the 830 patients in Poland’s SMA registry have either started taking Spinraza…
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
A plant used in traditional Chinese medicine boosts the production of functional survival motor neuron (SMN) protein — whose lack underlies the development of spinal muscular atrophy (SMA) — and ameliorates disease symptoms, a study in a mouse model finds. The results, “Improvement of Spinal Muscular Atrophy via Correction…
Babies with spinal muscular atrophy (SMA) treated with Zolgensma before experiencing symptoms continue to reach motor milestones in a normal timeframe, according to updated Phase 3 results. “With genetic testing in hand and presymptomatic treatment, we can look forward to a future in which age-appropriate motor milestone development…
A new global Phase 2/3 clinical trial will test whether higher doses of Spinraza (nusinersen) provide greater efficacy to spinal muscular atrophy (SMA) patients of all ages. Biogen aims to include 125 participants with infantile- or later-onset SMA in the DEVOTE study (NCT04089566), to be…
Inhibiting a specific signaling pathway — known as NF-κB — may help reduce chronic pain in spinal muscular atrophy (SMA), a new mouse study shows. The results of the study, “SMN deficiency causes pain hypersensitivity in a mild SMA mouse model through enhancing excitability of nociceptive dorsal…
Some $6.6 million in new Muscular Dystrophy Association (MDA) multiyear grants will support promising studies in an array of neuromuscular disorders, including spinal muscular atrophy (SMA). Through their work, the 25 award recipients hope to better understand disease mechanisms, build upon current treatments, advance the identification of new…
Transplanting a subset of neural stem cells (NSCs) to the spinal canal may be a potential therapy to delay disease progression in children with spinal muscular atrophy with respiratory distress type 1 (SMARD1), according to a preclinical study. The study, “CSF transplantation of a specific iPSC-derived…
Swallowing and speech problems are common in both children and adults with spinal muscular atrophy (SMA), and evident across disease types, a study reports. The most common bulbar complaints irrespective of SMA type are difficulties in biting, fatigue while chewing, choking, and a weak speaking voice. (Bulbar refers to…
The severity of motor problems in children at the time of diagnosis with spinal muscular atrophy (SMA) type 2 may help to predict likely disease progression, including the start of scoliosis and required ventilation, according to an Italian observational study. This is important because a better understanding SMA’s natural history — how…
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