Author:
Vockley Jerry,Burton Barbara,Jurecka Agnieszka,Ganju Jitendra,Leiro Beth,Zori Roberto,Longo Nicola
Funder
Acorda Therapeutics
National Institutes of Health
Subject
Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism
Reference127 articles.
1. Clinical trials in rare disease: challenges and opportunities;Augustine;J. Child Neurol.,2013
2. Applicability and added value of novel methods to improve drug development in rare diseases;Mitroiu;Orphanet J. Rare Dis.,2018
3. Guideline on Clinical Trials in Small Populations,2006
4. Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, Guidance for Industry, Draft guidance,2019
5. Expedited Programs for Serious Conditions – Drugs and Biologics, Guidance for Industry,2014