Clinical trial design for Friedreich ataxia - Where are we now and what do we need?
Author:
Affiliation:
1. Departments of Neurology and Pediatrics, Clinical Data Science GmbH, Basel, Switzerland
2. Division of Neurology, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA
Funder
not funded
Publisher
Informa UK Limited
Subject
Pharmacology (medical),Health Policy,Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
Link
https://www.tandfonline.com/doi/pdf/10.1080/21678707.2018.1449638
Reference76 articles.
1. Pharmacological therapeutics in Friedreich ataxia: the present state
2. Friedreich Ataxia
3. FRIEDREICH'S ATAXIA: A CLINICAL AND GENETIC STUDY OF 90 FAMILIES WITH AN ANALYSIS OF EARLY DIAGNOSTIC CRITERIA AND INTRAFAMILIAL CLUSTERING OF CLINICAL FEATURES
4. A 22-Year Follow-up Study of Long-term Cardiac Outcome and Predictors of Survival in Friedreich Ataxia
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