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Metadata

Name
Phase I/II Study of SRP-4053 in DMD Patients
Repository
ClinicalTrials.gov
Identifier
clinicaltrials:NCT02310906
Description
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability,
efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with
deletions amenable to exon 53 skipping.
Data or Study Types
clinical trial
Keywords
Exon 53, DMD, Exon Skipping, Pediatric, Duchenne muscular dystrophy, Ambulatory
Source Organization
Unknown
Access Conditions
available
Year
2014
Access Hyperlink
https://clinicaltrials.gov/ct2/show/NCT02310906

Distributions

  • Encoding Format: HTML ; URL: https://clinicaltrials.gov/ct2/show/results/NCT02310906
This project was funded in part by grant U24AI117966 from the NIH National Institute of Allergy and Infectious Diseases as part of the Big Data to Knowledge program. We thank all members of the bioCADDIE community for their valuable input on the overall project.