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WEB6 days ago · Standardized SMA newborn screening may aid early treatment. Substantial differences in newborn screening (NBS) practices for spinal muscular atrophy (SMA) across the U.S. could impact referral patterns or the timing …
With Zolgensma, Gene Therapy Takes Step Forward in Treating …
WEBMay 31, 2019 · Zolgensma‘s approval as the first gene therapy for treating all forms of spinal muscular atrophy (SMA) in children up to 2 years old is another step forward — an exceptional and far-reaching one — in the seeming revolution underway in treating more diseases, and an increasing number of rare ones, by correcting problems in the illness-causing gene itself.
Across August, Variety of Events Celebrate SMA Awareness Month
WEBAug 2, 2021 · Learn how to participate in various activities and events organized by Cure SMA and other groups to raise awareness and funds for spinal muscular atrophy (SMA) in August. Read stories from the SMA community, share facts and graphics, and get involved with advocacy issues.
HCP SMA News Today – The Web's Leading Resource For People …
WEBHigh-dose Spinraza treatment shown to safely benefit SMA infants. Treatment with an investigational high-dose regimen of Spinraza (nusinersen) was well tolerated and significantly improved motor function in infants with spinal muscular atrophy (SMA) relative to untreated patients, according to top-line data …
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 anthropomorphic measurements. 3 In younger children, a ...
SMA Type 2: Symptoms, Treatment, and More - SMA News Today
WEBFeb 2, 2023 · SMA type 2 is a rare genetic disease that causes muscle weakness and wasting in infancy. Learn about the symptoms, diagnosis, treatment options, and life expectancy of this intermediate form of spinal muscular atrophy.
Branaplam – HCP SMA News Today
WEB8. Palacino J, Swalley SE, Song C, et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat Chem Biol. Jul 2015;11(7):511-517. 9. Charnas L, Voltz E, Pfister C, et al. Safety and efficacy findings in the first-in-human trial (FIH) of the oral splice modulator branaplam in type 1 spinal muscular atrophy (SMA): interim results.
Spinal Muscular Atrophy: Cardiac Disease – HCP SMA News Today
WEBLearn how SMA affects the heart and increases the risk of congenital heart defects, ECG abnormalities and cardiac fibrosis. Find out the types of SMA and their association with cardiac pathology and the number of SMN2 gene copies.
Spinal muscular atrophy diagnosis and testing - SMA News Today
WEBJan 20, 2023 · Due to the progressive nature of spinal muscular atrophy (SMA) — a rare genetic disease caused by the progressive loss of motor neurons, the specialized nerve cells that control voluntary movements — an early diagnosis is critical. People living with SMA who receive a timely diagnosis can start receiving treatment immediately.
Oral SBMA therapy shows safety and tolerability in Phase 1 trial
WEBNov 15, 2023 · NIDO-361 is a small molecule that binds to the androgen receptor, correcting its abnormal function in spinal and bulbar muscular atrophy (SBMA). A Phase 1 trial showed it to be safe and well tolerated in healthy …