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 +5 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 +13 more sources
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 ...
Marleen Van Den Hauwe +2 more
exaly +6 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
Risdiplam Add-On Therapy Following Onasemnogene Abeparvovec in Children With Spinal Muscular Atrophy and 2 SMN2 Copies: A Multi-Center Case Series. [PDF]
Muscle NerveABSTRACT Introduction/Aims Three disease‐modifying therapies are approved for individuals with spinal muscular atrophy (SMA); however, data concerning the combination of these therapies remain limited. This study aimed to evaluate the safety and efficacy of add‐on risdiplam in children who had experienced clinical deterioration despite gene therapy ...
Stoltenburg C +4 more
europepmc +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).
Qiao Yue
exaly +3 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
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
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