A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies.

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Bibliographic Details
Title: A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies.
Authors: Wilk J; Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA.; Department of Mathematics, College of Sciences, University of Central Florida, Orlando, Florida, USA., Aggarwal V; Critical Path Institute (C-Path), Tucson, Arizona, USA., Pauley M; Critical Path Institute (C-Path), Tucson, Arizona, USA., Corey D; Critical Path Institute (C-Path), Tucson, Arizona, USA., Conrado DJ; e-Quantify LLC, La Jolla, California, USA., Lingineni K; Novartis Pharmaceutical Corporation, Cambridge, Massachusetts, USA., Morales JF; Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA.; Critical Path Institute (C-Path), Tucson, Arizona, USA., Yoon DY; Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA., Zhang Y; Critical Path Institute (C-Path), Tucson, Arizona, USA., Cui Z; Critical Path Institute (C-Path), Tucson, Arizona, USA., Burton J; Biogen, Cambridge, Massachusetts, USA., Larkindale J; Critical Path Institute (C-Path), Tucson, Arizona, USA., Ma SC; Critical Path Institute (C-Path), Tucson, Arizona, USA., Hovinga C; Critical Path Institute (C-Path), Tucson, Arizona, USA., Martinez T; Critical Path Institute (C-Path), Tucson, Arizona, USA., Romero K; Critical Path Institute (C-Path), Tucson, Arizona, USA., Belfiore-Oshan R; Critical Path Institute (C-Path), Tucson, Arizona, USA., Kim S; Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy, University of Florida, Orlando, Florida, USA.
Corporate Authors: Duchenne Regulatory Science Consortium (D‐RSC) and the CINRG DNHS investigators
Source: CPT: pharmacometrics & systems pharmacology [CPT Pharmacometrics Syst Pharmacol] 2025 Nov; Vol. 14 (11), pp. 1787-1796. Date of Electronic Publication: 2024 Nov 27.
Publication Type: Case Reports; Journal Article
Journal Info: Publisher: Wiley Country of Publication: United States NLM ID: 101580011 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2163-8306 (Electronic) Linking ISSN: 21638306 NLM ISO Abbreviation: CPT Pharmacometrics Syst Pharmacol Subsets: MEDLINE
Database: MEDLINE Ultimate
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Description
ISSN:2163-8306
DOI:10.1002/psp4.13281