Risdiplam: Therapeutic Effects and Tolerability in a Small Cohort of 6 Adult Type 2 and Type 3 SMA Patients (P11-11.018)

To describe the effect of Risdiplam on a cohort of 6 adult SMA patients using Motor Function Measure (MFM32) score as standardized scale to quantify motor improvements induced by therapy.

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Publication Details

Journal
Neurology
Published
2025-04-07
DOI
https://doi.org/10.1212/wnl.0000000000211899
Primary Topic
Neurogenetic and Muscular Disorders Research
Type
article
Field-Weighted Citation Impact
0.00
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article

Risdiplam: Therapeutic Effects and Tolerability in a Small Cohort of 6 Adult Type 2 and Type 3 SMA Patients (P11-11.018)

Edoardo Malfatti, Gianmarco Severa, Christophe Alimi-Ichola
Neurology
Neurogenetic and Muscular Disorders Research
article

Risdiplam: Therapeutic Effects and Tolerability in a Small Cohort of 6 Adult Type 2 and Type 3 SMA Patients (P11-11.018)

Edoardo Malfatti, Gianmarco Severa, Christophe Alimi-Ichola
article en

Abstract

To describe the effect of Risdiplam on a cohort of 6 adult SMA patients using Motor Function Measure (MFM32) score as standardized scale to quantify motor improvements induced by therapy.

NeurologyVol. 104(7_Supplement_1)
Paris-Est Sup (FR)
Openalex Percentile: Top 90%
Neurogenetic and Muscular Disorders Research
DOISourceOpenAlex
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Risdiplam: Therapeutic Effects and Tolerability in a Small Cohort of 6 Adult Type 2 and Type 3 SMA Patients (P11-11.018) — Edoardo Malfatti, Gianmarco Severa, et al. · Neurology (2025) | TGRS Research Map | TGRS