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Annals of Clinical and Translational Neurology|August 16, 2019
Measuring peripheral nerve involvement in Friedreich's ataxiaPeter D Creigh, Joan Mountain, Janet E Sowden, et al.
Orphanet Journal of Rare Diseases|January 24, 2018
Principles for interactions with biopharmaceutical companies: the development of guidelines for patient advocacy organizations in the field of rare diseasesSusan Stein, Elizabeth Bogard, Nicole Boice, et al.
Future Science OA|July 10, 2019
The current state of biomarker research for Friedreich's ataxia: a report from the 2018 FARA biomarker meetingIan A Blair, Jennifer Farmer, Steven Hersch, et al.
Muscle & Nerve|July 10, 2013
Cost of illness for neuromuscular diseases in the United StatesJane Larkindale, Wenya Yang, Paul F Hogan, et al.
Therapeutic Innovation & Regulatory Science|January 5, 2021
Effective Data Sharing as a Conduit for Advancing Medical Product DevelopmentStephen R Karpen, J Kael White, Ariana P Mullin, et al.
CPT: Pharmacometrics & Systems Pharmacology|December 8, 2021
Development of a model-based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophyKarthik Lingineni, Varun Aggarwal, Juan Francisco Morales, et al.
Plos One|May 15, 2020
Serum glutamate dehydrogenase activity enables early detection of liver injury in subjects with underlying muscle impairmentsShelli Schomaker, David Potter, Roscoe Warner, et al.
Therapeutic Innovation & Regulatory Science|June 6, 2022
Innovations in Therapy Development for Rare Diseases Through the Rare Disease Cures Accelerator-Data and Analytics PlatformJane Larkindale, Alexandre Betourne, Amanda Borens, et al.
Clinical and Translational Science|July 24, 2020
Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington's DiseaseAriana P Mullin, Diane Corey, Emily C Turner, et al.
Neurology|November 28, 2022
Patient-Reported Impact of Symptoms in Friedreich AtaxiaJamison Seabury, Danae Alexandrou, Nuran Dilek, et al.
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