Lessons from the ATTeST trial in ataxia telangiectasia
Author:
Publisher
Elsevier BV
Reference7 articles.
1. Ataxia telangiectasia: a review;Rothblum-Oviatt;Orphanet J Rare Dis,2016
2. Clinical trials in rare disease: challenges and opportunities;Augustine;J Child Neurol,2013
3. A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial;Crow;Trials,2018
4. Study design challenges and strategies in clinical trials for rare diseases: lessons learned from pantothenate kinase-associated neurodegeneration;Videnovic;Front Neurol,2023
5. Safety and efficacy of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia (ATTeST): a multicentre, randomised, double-blind, placebo-controlled phase 3 trial;Zielen;Lancet Neurol,2024
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