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Onasemnogene Abeparvovec Effective in Treatment of Older, Heavier Children with SMA

A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1. ....

United Kingdom , United States , Simon Dannhauer , Lancet Regional Health , Philadelphia Infant Test , Neuromuscular Disorders , Hammersmith Infant Neurological Examination , Revised Hammersmith Scale , Spinal Muscular Atrophy , Sma Type 1 , Onasemnogene Abeparvovec ,

Risdiplam May Be Safe and Effective in SMA Care

Risdiplam appears to be an effective therapeutic option for patients with spinal muscular atrophy, leading to improved motor function, a study found. ....

United States , Philadelphia Infant Test , Neuromuscular Disorders , Hammersmith Infant Neurological Exam , Motor Function Measure , Revised Upper Limb Module , Hammersmith Functional Motor Scale ,

Spinraza benefits SMA children not helped by Zolgensma: Interim data

Spinraza benefits SMA children not helped by Zolgensma: Interim data
smanewstoday.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from smanewstoday.com Daily Mail and Mail on Sunday newspapers.

United States , Maha Radhakrishnan , Novarti Zolgensma , World Health Organization , Research Clinical Care Meeting , Clinical Care Meeting , Philadelphia Infant Test , Neuromuscular Disorders , Hammersmith Functional Motor Scale Expanded , Revised Upper Limb , Alcyone Therapeutics ,

Current treatments improve motor function in SMA type 1 children across Spain | Study examines effects of current SMA therapies on 51 children in Spain

Current treatments improve motor function in SMA type 1 children across Spain | Study examines effects of current SMA therapies on 51 children in Spain
smanewstoday.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from smanewstoday.com Daily Mail and Mail on Sunday newspapers.

United States , National Sample Of Spanish Children , Functional Characteristics , New Phenotype , National Sample , Spanish Children , Cross Sectional Study , Hammersmith Infant Neurological Examination , Philadelphia Infant Test , Neuromuscular Disorders , Hammersmith Functional Motor Scale Expanded , Upper Limb Module ,

Disease Modifying Therapies for Children with Spinal Atrophy

An update on the emerging evidence of the effects and safety of disease modifying therapies for the management of children with spinal muscular atrophy (SMA). ....

United States , Noord Holland , Nordrhein Westfalen , Nat Genet , Hansa Biopharma , Petzolda Neurofilament , Clin Neurophysiol , Sanofi Genzyme , Thomas Sejersen , Neuromuscul Disord , Pediatr Pulmonol , Dement Neuropsychol , Zetterbergh Neurofilament , Dunaway Young , Dhillons Risdiplam , World Muscle Society Virtual Congress , Charles River Associates , European Medicines Agency , Us Food Drug Administration , Lancet Child Adolesc Health , Sarepta Therapeutics , Clin North , Treat Options , Opin Investig , Child Adolesc , Orphanetj Rare ,