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21
datasets available to search
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Dataset results
21 results for “risdiplam”
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
ClinicalTrials.gov study NCT03779334. IPD Sharing: YES. Countries: 7. Publications: 1.
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
ClinicalTrials.gov study NCT03032172. IPD Sharing: YES. Countries: 9. Publications: 1.
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
ClinicalTrials.gov study NCT03988907. IPD Sharing: NO. Countries: 1. Publications: 1.
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
ClinicalTrials.gov study NCT02913482. IPD Sharing: YES. Countries: 16. Publications: 5.
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
ClinicalTrials.gov study NCT02908685. IPD Sharing: Not stated. Countries: 16. Publications: 2.
Data from: Adverse events associated with risdiplam use
<p>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 current study was to examine the AEs associated with risdiplam use based on real-world data obtained from the US Food and Drug Administration Adverse Event Reporting System (FAERS) database. More specifically, we examined incidents reported between the third quarter of 2020 and the second quarter of 2023. The imbalance of risdiplam-related AEs was evaluated by computing the reporting odds ratio. A total of 5,406,334 reports were thoroughly reviewed. By removing duplicate reports, we identified 1588 reports in which risdiplam was the main suspected drug whose use was accompanied by 3470 associated AEs. Among the included AEs, 703 were categorized as serious and 885 as non-serious. Risdiplam use induced AEs across 18 organ systems, resulting in 130 positive signals. Notably, we detected new AE signals, including cardiac arrest, nephrolithiasis, tachycardia, loss of libido, and elevated hepatic enzyme activities; however, no ophthalmologic toxicity was reported. Although these new adverse reaction signals associated with risdiplam have been defined, long-term clinical studies are needed to confirm these findings. Nevertheless, our findings provide a valuable reference for improving the clinical management of SMA.</p>
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
ClinicalTrials.gov study NCT05156320. IPD Sharing: NO. Countries: 9. Publications: 1.
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
ClinicalTrials.gov study NCT05522361. IPD Sharing: NO. Countries: 1. Publications: 3.
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers
ClinicalTrials.gov study NCT02633709. IPD Sharing: Not stated. Countries: 1. Publications: 1.
Data from: Adverse events associated with risdiplam use
Open the record for dataset details and reuse information.
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
ClinicalTrials.gov study NCT05861986. IPD Sharing: NO. Countries: 4. Publications: 0.
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
ClinicalTrials.gov study NCT05861999. IPD Sharing: NO. Countries: 6. Publications: 0.
A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepa
ClinicalTrials.gov study NCT03920865. IPD Sharing: NO. Countries: 1. Publications: 0.
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
ClinicalTrials.gov study NCT04256265. IPD Sharing: Not stated. Countries: 1. Publications: 1.
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
ClinicalTrials.gov study NCT05808764. IPD Sharing: YES. Countries: 8. Publications: 0.
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
ClinicalTrials.gov study NCT05232929. IPD Sharing: YES. Countries: 2. Publications: 0.
A Study Investigating the Excretion Balance, Pharmacokinetics and Metabolism of a Single Oral Dose of [14C]-Labeled Risdiplam (RO7034067) in Healthy Male Participants
ClinicalTrials.gov study NCT03036501. IPD Sharing: Not stated. Countries: 1. Publications: 0.
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
ClinicalTrials.gov study NCT05115110. IPD Sharing: YES. Countries: 12. Publications: 0.
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
ClinicalTrials.gov study NCT04718181. IPD Sharing: NO. Countries: 1. Publications: 0.
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
ClinicalTrials.gov study NCT05067790. IPD Sharing: YES. Countries: 9. Publications: 0.
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Allen Brain Atlas
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International Brain Laboratory public data
The International Brain Laboratory public data releases expose standardized mouse decision-making experiments, including Neuropixels recordings, widefield calcium imaging, behavior, and session metadata accessed through the ONE API.
OpenNeuro
OpenNeuro is a free, open platform for sharing neuroimaging datasets, with public search, dataset pages, and download paths for web, S3, DataLad, and the OpenNeuro CLI.