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Authors
Tandon, P. K. https://orcid.org/0000-0001-7753-8923
Kakkis, Emil D.
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Clinical Trials BETA
Clinical trials referenced in this document:
nct00912925 at ClinicalTrials.govnct00146770 at ClinicalTrials.govDocuments that mention this clinical trial
The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases
https://doi.org/10.1186/s13023-021-01805-5
nct00067470 at ClinicalTrials.govDocuments that mention this clinical trial
The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases
https://doi.org/10.1186/s13023-021-01805-5
nct00104234 at ClinicalTrials.govDocuments that mention this clinical trial
The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases
https://doi.org/10.1186/s13023-021-01805-5
nct00069641 at ClinicalTrials.govDocuments that mention this clinical trial
The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases
https://doi.org/10.1186/s13023-021-01805-5
nct02230566 at ClinicalTrials.govDocuments that mention this clinical trial
The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases
https://doi.org/10.1186/s13023-021-01805-5
nct02377921 at ClinicalTrials.govDocuments that mention this clinical trial
The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases
https://doi.org/10.1186/s13023-021-01805-5
Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials
https://doi.org/10.1007/s40262-018-0721-y
nct02432144 at ClinicalTrials.govDocuments that mention this clinical trial
The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases
https://doi.org/10.1186/s13023-021-01805-5
Elevated Plasma Free Sialic Acid Levels in Individuals with Reduced Glomerular Filtration Rates
https://doi.org/10.34067/kid.0002122020
Long-term evaluation parameters in GNE myopathy: a 5-year observational follow-up natural history study
https://doi.org/10.1136/bmjno-2022-000362
Documents that mention this clinical trial
The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases
https://doi.org/10.1186/s13023-021-01805-5
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Funding
Funding for this research was provided by:
Ultragenyx Pharmaceutical
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Article History
Received: 8 September 2020
Accepted: 30 March 2021
First Online: 19 April 2021
Declarations
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: All reported studies involving human participants were approved by each clinical site’s institutional review board or independent ethics committee. All parents/legal guardians and adult participants provided written informed consent to participate in each study before any study specific procedures were performed.
: Not applicable.
: PKT and EDK are employees and shareholders of Ultragenyx Pharmaceutical Inc.
