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Authors
Cleary, Yumi https://orcid.org/0000-0002-9703-7269
Kletzl, Heidemarie
Grimsey, Paul
Heinig, Katja
Ogungbenro, Kayode https://orcid.org/0000-0003-2446-6895
Silber Baumann, Hanna Elisabeth
Frey, Nicolas
Aarons, Leon https://orcid.org/0000-0003-2704-6570
Galetin, Aleksandra
Gertz, Michael
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Clinical Trials BETA
Clinical trials referenced in this document:
nct02633709 at ClinicalTrials.govnct03032172 at ClinicalTrials.govDocuments that mention this clinical trial
Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug–Drug Interactions in Children
https://doi.org/10.1007/s40262-023-01241-7
nct02908685 at ClinicalTrials.govDocuments that mention this clinical trial
Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug–Drug Interactions in Children
https://doi.org/10.1007/s40262-023-01241-7
Safety update: risdiplam clinical trial program for spinal muscular atrophy
https://doi.org/10.1136/jnnp-2024-abn.159
185 JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with SMA receiving risdiplam
https://doi.org/10.1136/jnnp-2022-abn2.229
Safety update: risdiplam clinical trial programme for spinal muscular atrophy (SMA)
https://doi.org/10.1136/jnnp-2023-abn.178
182 Pooled safety data from the risdiplam clinical development programme
https://doi.org/10.1136/jnnp-2022-abn2.226
JEWELFISH: 24-month safety and pharmacodynamic data in non-treatment- naïve patients with spinal muscular atrophy (SMA)
https://doi.org/10.1136/jnnp-2023-abn.177
nct02913482 at ClinicalTrials.govDocuments that mention this clinical trial
Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug–Drug Interactions in Children
https://doi.org/10.1007/s40262-023-01241-7
Safety update: risdiplam clinical trial program for spinal muscular atrophy
https://doi.org/10.1136/jnnp-2024-abn.159
185 JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with SMA receiving risdiplam
https://doi.org/10.1136/jnnp-2022-abn2.229
SUNFISH: 4-year efficacy and safety data of risdiplam in types 2 and 3 SMA
https://doi.org/10.1136/jnnp-2023-abn.179
SUNFISH Part 2: 24-month efficacy and safety of risdiplam in Type 2/3 SMA
https://doi.org/10.1136/jnnp-2022-abn.309
255 SUNFISH part 2: risdiplam in type 2 and type 3 SMA
https://doi.org/10.1136/jnnp-2022-abn.282
Safety update: risdiplam clinical trial programme for spinal muscular atrophy (SMA)
https://doi.org/10.1136/jnnp-2023-abn.178
187 SUNFISH Part 2: 24-month efficacy of risdiplam compared with external control comparators
https://doi.org/10.1136/jnnp-2022-abn2.231
182 Pooled safety data from the risdiplam clinical development programme
https://doi.org/10.1136/jnnp-2022-abn2.226
131 Pooled safety data from the risdiplam clinical trial development programme
https://doi.org/10.1136/jnnp-2022-abn.456
JEWELFISH: 24-month safety and pharmacodynamic data in non-treatment- naïve patients with spinal muscular atrophy (SMA)
https://doi.org/10.1136/jnnp-2023-abn.177
Post hoc analysis of the SMA independence scale-upper limb module using SUNFISH Part 2 data
https://doi.org/10.1136/jnnp-2024-abn.177
188 SUNFISH 3-year efficacy and safety of risdiplam in types 2 and 3 SMA
https://doi.org/10.1136/jnnp-2022-abn2.232
Correlation of individual SMN protein with observed efficacy in the risdiplam SMA clinical trials
https://doi.org/10.1136/jnnp-2024-abn.184
nct03988907 at ClinicalTrials.govDocuments that mention this clinical trial
Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug–Drug Interactions in Children
https://doi.org/10.1007/s40262-023-01241-7
Safety update: risdiplam clinical trial program for spinal muscular atrophy
https://doi.org/10.1136/jnnp-2024-abn.159
256 FIREFISH part 2: risdiplam efficacy and safety in type 1 SMA
https://doi.org/10.1136/jnnp-2022-abn.283
Safety update: risdiplam clinical trial programme for spinal muscular atrophy (SMA)
https://doi.org/10.1136/jnnp-2023-abn.178
182 Pooled safety data from the risdiplam clinical development programme
https://doi.org/10.1136/jnnp-2022-abn2.226
131 Pooled safety data from the risdiplam clinical trial development programme
https://doi.org/10.1136/jnnp-2022-abn.456
JEWELFISH: 24-month safety and pharmacodynamic data in non-treatment- naïve patients with spinal muscular atrophy (SMA)
https://doi.org/10.1136/jnnp-2023-abn.177
Correlation of individual SMN protein with observed efficacy in the risdiplam SMA clinical trials
https://doi.org/10.1136/jnnp-2024-abn.184
Documents that mention this clinical trial
Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug–Drug Interactions in Children
https://doi.org/10.1007/s40262-023-01241-7
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Funding
Funding for this research was provided by:
F. Hoffmann-La Roche
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Article History
Accepted: 19 March 2023
First Online: 6 May 2023
Declarations
:
: Yumi Cleary, Heidemarie Kletzl, Paul Grimsey, Katja Heinig, Hanna E. Silber Baumann, Nicolas Frey and Michael Gertz are employees of F. Hofmann-La Roche, Ltd, and hold stocks/stock options with F. Hoffmann-La Roche, Ltd. Kayode Ogungbenro, Leon Aarons and Aleksandra Galetin declared no competing interests for this work.
: All relevant study documents were approved by the Institutional Review Board and all subjects or their guardians signed the informed consent prior to enrolment. The studies were conducted in full conformance with the principles of the Declaration of Helsinki and its later amendments or comparable ethical standards.
: Informed consent was obtained from all individual participants included in the studies discussed.
: This study was funded by F. Hoffmann-La Roche Ltd. Open access publication of this manuscript was sponsored by F. Hoffmann-La Roche, Ltd.
: Qualified researchers may request access to individual patient-level data through the clinical study data request platform (). Further details on Roche’s criteria for eligible studies are available at . For further details on Roche’s Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see .
