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Spinraza led to motor gains for 3 SMA adults in small Japanese study| Spinraza s benefits seen in some adult SMA patients but more study needed

Spinraza led to motor gains for 3 SMA adults in small Japanese study| Spinraza s benefits seen in some adult SMA patients but more study needed
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Japan generalNagara medical centerHammersmith functional motor scale

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

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

Cognition in Patients With Spinal Muscular Atrophy Tied to Disease Severity

Cognitive function was associated with disease severity and motor impairment in patients with type 3 SMA, suggesting the presence of intrinsic brain pathology.

United statesMini mental state examinationHammersmith functional motor scaleRevised upper limb moduleMinute walk testRaven progressive matricesPhonetic fluencyDigit spanStroop color word testTrail making testsBoston naming testSemantic fluencyRey osterrieth complex figure testDigit spin forwardRey auditory verbal learning test

New Roche data for Evrysdi show improved motor function in pre-symptomatic babies after one year and confirm safety profile in previously treated people with spinal muscular atrophy (SMA)

Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new interim data from two studies of Evrysdi® (risdiplam); JEWELFISH and RAINBOWFISH. Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in people aged 1 to 60 years who have been previously treated with another .

New yorkUnited statesColumbia universityLevi garrawayClaudia chiribogaDepartment of neurologyToddler developmentDrug administrationResearch clinical care meetingHead of global product developmentColumbia university medical centerEuropean medicines agencyRoche groupPharmaceuticals industryClinical care meetingChief medical officer