January 8, 2021 Effective Data Sharing as a Conduit for Advancing Medical Product Development Karpen SR, White JK, Mullin AP, O'Doherty I, Hudson LD, Romero K, Sivakumaran....
June 14, 2020 Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington’s Disease
May 14, 2020 Serum Glutamate Dehydrogenase Activity Enables Early Detection of Liver Injury in Subjects with Underlying Muscle Impairments
August 14, 2019 Conrado et al. 2019 Towards Regulatory Endorsement Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in...
December 1, 2017 Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular Dystrophy
October 26, 2017 226th ENMC International Workshop: Towards Validated and Qualified Biomarkers for Therapy Development for Duchenne Muscular Dystrophy
April 18, 2024 The Duchenne Regulatory Science Consortium – A public-private partnership to facilitate sharing of clinical data for the development of regulatory tools to accelerate drug development (Abstract)
April 18, 2024 The TREAT-NMD Advisory Committee for Therapeutics (TACT): An Innovative de-Risking Model to Foster Orphan Drug Development