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Cerebellum (London, England)|December 2, 2021
The Responsiveness of Gait and Balance Outcomes to Disease Progression in Friedreich AtaxiaSarah C Milne, Seok Hun Kim, Anna Murphy, et al.CPT: Pharmacometrics & Systems Pharmacology|November 27, 2024
A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studiesJordan Wilk, Varun Aggarwal, Mike Pauley, et al.Neurology. Clinical Practice|August 30, 2023
Friedreich's Ataxia-Health Index: Development and Validation of a Novel Disease-Specific Patient-Reported Outcome MeasureJamison Seabury, Spencer Rosero, Anika Varma, et al.Therapeutic Advances in Rare Disease|August 2, 2023
The IRDiRC Chrysalis Task Force: making rare disease research attractive to companiesKatherine L Beaverson, Daria Julkowska, Mary Catherine V Letinturier, et al.Journal of Pharmacokinetics and Pharmacodynamics|May 26, 2019
Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophyDaniela J Conrado, Jane Larkindale, Alexander Berg, et al.Plos Currents|February 24, 2017
Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular DystrophyJane Larkindale, Richard Abresch, Enrique Aviles, et al.Human Gene Therapy|February 6, 2015
Perspectives on best practices for gene therapy programsThomas R Cheever, Dale Berkley, Serge Braun, et al.Pageof 3