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WEBTreatment with Spinraza (nusinersen) generally stabilized or improved motor function for older patients with spinal muscular atrophy (SMA), according to a review and meta-analysis of previously published observational studies. The new analysis, which spanned a treatment period of up to two years, involved adults and adolescents with….
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WEBSep 18, 2024 · The use of ultrasound to guide the administration of Spinraza (nusinersen) injection into the spinal canals of adults with spinal muscle atrophy (SMA) significantly reduced the number of needle redirections compared with a standard, landmark-based approach, according to a clinical trial. Among patients with …
Spinal Muscular Atrophy: Nutrition and Supplementation
WEBThe nutritional management of patients with progressive neuromuscular diseases like Spinal Muscular Atrophy (SMA) is complex and is achieved optimally with an individualized approach from a multidisciplinary care team. 1,2 Patients with SMA need nutritional monitoring at least annually with assessment of caloric intake and standard ...
Spinal Muscular Atrophy and Genetics – SMA News Today
WEBDid You Know? SMA has a wide spectrum of severity that is mostly explained by differences in the number of copies of the backup gene, SMN2.However, up to 40% of the variability in disease severity is not associated with the number of SMN2 copies. Possible factors may be genes that produce proteins that directly interact with the SMN protein or are connected to its proper function.
FAQs About Evrysdi (Risdiplam) - SMA News Today
WEBAug 7, 2020 · FAQs About Evrysdi (Risdiplam) by Grace Frank August 7, 2020. Evrysdi (risdiplam) is an oral liquid treatment approved in the U.S. for infants through adults with spinal muscular atrophy (SMA) — patients age 2 months and older with any disease type. Roche and Genentech developed Evrysdi in collaboration with …
Spinal Muscular Atrophy: Cardiac Disease – HCP SMA News Today
WEBSpinal Muscular Atrophy (SMA) is an inherited neurodegenerative disease, 1 but it is increasingly clear that the pathology of SMA extends beyond ventral horn spinal cord neurons and includes the heart among other organs. 2 In patients with SMA, tissue levels of SMN1 protein are reduced in heart tissues, 3 and numerous mouse models of SMA exhibit cardiac developmental abnormalities which ...
Warning of TMA, Blood Disorder, Added to Zolgensma's Safety Label
WEBMay 10, 2021 · Novartis Gene Therapies has added thrombotic microangiopathy (TMA) — a rare condition characterized by blood abnormalities — to the list of safety concerns with use of Zolgensma in children with spinal muscular atrophy (SMA). This decision was driven by reports of six confirmed cases of TMA within one to two weeks of the gene therapy’s ...
Spinal Muscular Atrophy: Electromyography - SMA News Today
WEBSpinal Muscular Atrophy (SMA) is a progressive neuromuscular disease associated with typically proximal muscle weakness and atrophy due to degeneration of the anterior horn cells of the spinal cord. 1 Prior to genetic testing as the gold standard diagnostic method for SMA, electromyography (EMG) was used widely to diagnose SMA. 2,3 EMG still has a role in the diagnostic confirmation of motor ...
SMA Type 2: Symptoms, Treatment, and More - SMA News Today
WEBFeb 2, 2023 · Muscle weakness also can affect muscles in the chest, throat, and mouth, giving rise to other SMA type 2 symptoms that affect daily living. Such symptoms include: problems eating, due to weakness in the muscles used to chew and swallow. breathing and coughing difficulties resulting from chest muscle weakness, which can …
FDA Approves Evrysdi, First Oral Treatment for All SMA Types
WEBAug 7, 2020 · The U.S. Food and Drug Administration (FDA) has approved Evrysdi — formerly known as risdiplam — as the first oral and at-home treatment for adults, children, and infants 2 months of age and older with all types of spinal muscular atrophy (SMA). With this action, Evrysdi — marketed by Genentech, a subsidiary of Roche — is now the third ...