Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study [PDF]
Neurology and Therapy, 2023 Introduction Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier for the treatment of patients with spinal muscular atrophy (SMA). The JEWELFISH study (NCT03032172) was designed to assess the safety, tolerability, pharmacokinetics (PK ...
Claudia A. Chiriboga +16 more
doaj +7 more sources
Risdiplam in Type 1 Spinal Muscular Atrophy [PDF]
New England Journal of Medicine, 2021 Type 1 spinal muscular atrophy is a rare, progressive neuromuscular disease that is caused by low levels of functional survival of motor neuron (SMN) protein. Risdiplam is an orally administered, small molecule that modifies SMN2 pre-messenger RNA splicing and increases levels of functional SMN protein.We report the results of part 1 of a two-part ...
Giovanni Baranello +2 more
exaly +12 more sources
A Convenient, Pd-Free Approach to the Synthesis of Risdiplam [PDF]
MoleculesSeveral approaches to the synthesis of risdiplam, a pharmacologically relevant pyridopyrimidinone derivative, have been recently reported. However, most of these routes rely exclusively on palladium-catalyzed, cross-coupling reactions and involve low ...
Georgiy Korenev +7 more
doaj +3 more sources
Treatment of risdiplam after nusinersen continuously improves upper limb motor function in spinal muscular atrophy patients: a multicenter experience [PDF]
Frontiers in PediatricsIntroductionSome individuals with spinal muscular atrophy (SMA) transition from nusinersen to risdiplam during disease-modifying therapy (DMT) due to factors such as treatment convenience, economic considerations, and adverse events (AEs).
Xi Cheng +6 more
doaj +2 more sources
Comprehensive Risdiplam Synthesis Overview: From Cross-Coupling Reliance to Complete Palladium Independence [PDF]
MoleculesRisdiplam is the first approved small-molecule therapy for spinal muscular atrophy (SMA), a severe, progressive neuromuscular disorder. In addition to its clinical significance, risdiplam is of a great interest for organic and medicinal chemistry due to ...
Georgiy Korenev +2 more
doaj +2 more sources
Summary of Research: Fertility Outcomes in Risdiplam-Treated Male Patients with Spinal Muscular Atrophy: A Multicenter Case Series [PDF]
Neurology and TherapyThis Summary of Research summarizes a previously published original article, “Fertility Outcomes in Risdiplam-Treated Male Patients with Spinal Muscular Atrophy: A Multicenter Case Series.” Risdiplam (EVRYSDI®) is a medication approved for the treatment ...
Shelley Coskery +3 more
doaj +2 more sources
Effectiveness and safety of Risdiplam for types 1–3 spinal muscular atrophy in a single center [PDF]
BMC NeurologyBackground Risdiplam has demonstrated efficacy in various types of 5q-spinal muscular atrophy (SMA) during clinical trials, yet real-world data remain limited.
Xiaomei Zhu +6 more
doaj +2 more sources
A real-world, multicentre, epidemiological study in Czech and Slovak adults with spinal muscular atrophy treated with risdiplam [PDF]
Scientific ReportsReal-world evidence about risdiplam therapy in adults with 5q spinal muscular atrophy (SMA) remains limited to outcomes in small cohorts, heterogeneous endpoints, and a short follow-up.
Olesja Parmova +10 more
doaj +2 more sources
Brain Sciences, 2023 Objective: We performed a systematic review and meta-analysis of the efficacy and safety of nusinersen and risdiplam in the treatment of spinal muscular disease (SMA).
Yue Qiao, Yuewei Chi, Jian Gu, Ying Ma
doaj +3 more sources
Diverging Safety Signals: A Trend Analysis of Suspected Adverse Drug Reactions Reporting for Spinal Muscular Atrophy Therapies in the European Union [PDF]
Neurology InternationalBackground/Objectives: The approval of disease-modifying therapies has significantly improved outcomes for patients with spinal muscular atrophy (SMA), yet their long-term safety profiles remain under continuous evaluation.
Andrej Belančić +4 more
doaj +2 more sources