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PDFJun 30, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy
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PDFJun 30, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy
CureSMA FIREFISH Survival Presentation 2019: FIREFISH Part 1: Survival, ventilation and swallowing ability in infants with Type 1 spinal muscular atrophy (SMA) receiving risdiplam (RG7916)
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PDFJun 30, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy
CureSMA SUNFISH Presentation 2019: Update from SUNFISH Part 1: Safety, tolerability and PK/PD from the dose-finding study, including exploratory efficacy data, in patients with type 2 or 3 spinal muscular atrophy (SMA) treated with risdiplam (RG7916)
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PDFJun 28, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy
CureSMA JEWELFISH Poster 2019: JEWELFISH: Risdiplam (RG7916) increased survival of motor neuron (SMN) protein levels in patients with spinal muscular atrophy (SMA) that have previously received therapies targeting SMN2 splicing, or olesoxime
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PDFJun 28, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy
CureSMA FIREFISH Modified Bayley Poster 2019: Adaptation of the gross motor subscale of the Bayley Scales of Infant and Toddler Development, third edition (BSID-III) simplifies assessment for infants with Type 1 symptomatic spinal muscular atrophy (SMA)
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PDFJun 28, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy
CureSMA MFM 32 Poster 2019: Validity and reliability of the Motor Function Measure (MFM32) in children with neuromuscular disorders, and in individuals with Type 2 and non-ambulant Type 3 spinal muscular atrophy (SMA)
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PDFJun 28, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy
CureSMA Rasch Mapping Poster 2019: Using Rasch analysis to estimate thresholds associated with gain/loss of daily function on the Motor Function Measure (MFM)
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PDFJun 28, 2019 | Spinal Muscular Atrophy Cure SMA 2019 Spinal Muscular Atrophy
CureSMA Conceptual Modelling Poster 2019:Qualitative research to explore the patient and caregiver-reported experience of symptoms and impacts in spinal muscular atrophy (SMA) Types 2 and 3 - the development of a conceptual model