Scientists have developed a fast, efficient and economical method to create viral delivery vectors used in gene therapy to deliver modified versions of genes to treat disorders caused by genetic defects, including spinal muscular atrophy (SMA). The findings were reported in the study, “Production of adeno-associated virus…
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People with spinal muscular atrophy (SMA) type 1 may have an increased risk of impaired kidney function, a small study suggests. The study’s findings highlight the need to further evaluate non-muscular conditions associated with SMA type 1 and to identify co-adjuvant therapies to manage them. The research was published…
In an unprecedented decision reached Sept. 26 on the eve of Rosh Hashana — the Jewish New Year — Israel’s top health insurer, Clalit, said it would cover the $4.25 million cost of one-time Zolgensma gene therapy for two Israeli babies born with spinal muscular atrophy (SMA).
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…
