JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam
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DOI: 10.1007/s00415-024-12318-z
Abstract: Risdiplam is a once-daily oral, survival of motor neuron 2 (SMN2) splicing modifier approved for the treatment of spinal muscular atrophy (SMA). JEWELFISH (NCT03032172) investigated the safety, tolerability, pharmacokinetics (PK), and PK/pharmacodynamic (PD) relationship of… read more here.
Keywords: treatment; non treatment; patients sma; risdiplam ... See more keywords
Risdiplam utilization, adherence, and associated health care costs for patients with spinal muscular atrophy: a United States retrospective claims database analysis
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DOI: 10.1186/s13023-024-03399-0
Abstract: Spinal muscular atrophy (SMA) is a genetic neuromuscular disease associated with progressive loss of motor function. Risdiplam, a daily oral therapy, was approved in the United States for the treatment of SMA. Risdiplam’s effectiveness depends… read more here.
Keywords: risdiplam; care costs; type; adherence ... See more keywords
Longitudinal efficacy of risdiplam treatment in Chinese children with spinal muscular atrophy
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DOI: 10.1186/s13023-025-03929-4
Abstract: As the latest drug available for the treatment of spinal muscular atrophy (SMA), real-world research data on risdiplam are still lacking. The purpose of this study was to supplement the real-world data in SMA children… read more here.
Keywords: spinal muscular; treatment; risdiplam; muscular atrophy ... See more keywords
Exploration of adverse events associated with risdiplam use: Retrospective cases from the US Food and Drug Administration Adverse Event Reporting System (FAERS) database
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DOI: 10.1371/journal.pone.0298609
Abstract: Risdiplam is a new drug for treating spinal muscular atrophy (SMA). However, pharmacovigilance analyses are necessary to objectively evaluate its safety—a crucial step in preventing severe adverse events (AEs). Accordingly, the primary objective of the… read more here.
Keywords: adverse events; risdiplam; associated risdiplam; drug ... See more keywords
A Convenient, Pd-Free Approach to the Synthesis of Risdiplam
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DOI: 10.3390/molecules30163375
Abstract: Several 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-yielding intermediates, which limit their scalability… read more here.
Keywords: risdiplam; synthesis; free approach; synthesis risdiplam ... See more keywords