A single injection of Zolgensma into the spinal canal (intrathecal) can provide a clinically meaningful response in motor function gains among young children, 2 to 5 years old, who have spinal muscular atrophy (SMA) type 2, recent trial data show. Half of these older patients…
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SRK-015, an experimental treatment for spinal muscular atrophy (SMA), continues to produce promising results in preclinical studies and in a Phase 1 trial of healthy volunteers, its developer, Scholar Rock, has announced. “We are excited for the potential role of SRK-015 as the first muscle-directed therapy…
Risdiplam continues to show promise as a safe and effective therapy for people with spinal muscular atrophy (SMA) types 1, 2 and 3, according to the latest results from ongoing clinical trials. Data from the FIREFISH (NCT02913482), SUNFISH (NCT02908685), and JEWELFISH (NCT03032172) clinical trials were presented at the…
Chinese researchers have developed a fast, low-cost, and non-invasive maternal blood test for prenatal screening of spinal muscular atrophy (SMA). This method may be a safer alternative to traditional invasive prenatal screenings for families with a history of SMA and other high-risk populations, with the ability to identify affected…
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…
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…
