SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe
Authors
Keywords
Spinal muscular atrophy, Clinical trials, Preclinical drug selection, Phase III trial planning, Patient registries, Neonatal trials, Newborn screening, Standards of care, European regulations, Efficacy biomarkers
Find the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
SearchAsk a Question. Answer a Question.
Quickly pose questions to the entire community. Debate answers and get clarity on the most important issues facing researchers.
Get Started