The art and science of choosing efficacy endpoints for rare disease clinical trials
Author:
Affiliation:
1. Editas MedicineCambridgeMassachusetts
2. Division of GeneticsBoston Children's HospitalBostonMassachusetts
3. Department of PediatricsHarvard Medical SchoolBostonMassachusetts
Publisher
Wiley
Subject
Genetics(clinical),Genetics
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1002/ajmg.a.38629
Reference62 articles.
1. Alternative designs for clinical trials in rare diseases
2. Aldurazyme [laronidase] US package insert. BioMarin/Genzyme LLC. Novato CA/Cambridge MA; April2003.
3. Lung Function Testing: Selection of Reference Values and Interpretative Strategies
4. Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease
5. Evaluation of 11C-GSK189254 as a Novel Radioligand for the H3 Receptor in Humans Using PET
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