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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 kingdomUnited statesSimon dannhauerLancet regional healthPhiladelphia infant testNeuromuscular disordersHammersmith infant neurological examinationRevised hammersmith scaleSpinal muscular atrophySma type 1Onasemnogene 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 statesPhiladelphia infant testNeuromuscular disordersHammersmith infant neurological examMotor function measureRevised upper limb moduleHammersmith functional motor scale

Spinraza benefits SMA children not helped by Zolgensma: Interim data

Spinraza benefits SMA children not helped by Zolgensma: Interim data
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United statesMaha radhakrishnanNovarti zolgensmaWorld health organizationResearch clinical care meetingClinical care meetingPhiladelphia infant testNeuromuscular disordersHammersmith functional motor scale expandedRevised upper limbAlcyone 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
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United statesNational sample of spanish childrenFunctional characteristicsNew phenotypeNational sampleSpanish childrenCross sectional studyHammersmith infant neurological examinationPhiladelphia infant testNeuromuscular disordersHammersmith functional motor scale expandedUpper 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 statesNoord hollandNordrhein westfalenNat genetHansa biopharmaPetzolda neurofilamentClin neurophysiolSanofi genzymeThomas sejersenNeuromuscul disordPediatr pulmonolDement neuropsycholZetterbergh neurofilamentDunaway youngDhillons risdiplamWorld muscle society virtual congress