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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 General , Nagara Medical Center , Hammersmith 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 States , Philadelphia Infant Test , Neuromuscular Disorders , Hammersmith Infant Neurological Exam , Motor Function Measure , Revised Upper Limb Module , Hammersmith 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 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 ,

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 States , Mini Mental State Examination , Hammersmith Functional Motor Scale , Revised Upper Limb Module , Minute Walk Test , Raven Progressive Matrices , Phonetic Fluency , Digit Span , Stroop Color Word Test , Trail Making Tests , Boston Naming Test , Semantic Fluency , Rey Osterrieth Complex Figure Test , Digit Spin Forward , Rey 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 York , United States , Columbia University , Levi Garraway , Claudia Chiriboga , Department Of Neurology , Toddler Development , Drug Administration , Research Clinical Care Meeting , Head Of Global Product Development , Columbia University Medical Center , European Medicines Agency , Roche Group , Pharmaceuticals Industry , Clinical Care Meeting , Chief Medical Officer , Global Product , Orphan Drug Designation , Gross Motor Scale , Bayley Scales , Third Edition , Motor Function Measure , World Health Organization Model Lists , Essential Medicines , Dow Jones Sustainability Indices , Chugai Pharmaceutical ,