SMA-Europe workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe

17Citations
Citations of this article
27Readers
Mendeley users who have this article in their library.

This article is free to access.

Abstract

Spinal muscular atrophy (SMA) is the most common lethal recessive disease in childhood, and there is currently no effective treatment to halt disease progression. The translation of scientific advances into effective therapies is hampered by major roadblocks in clinical trials, including the complex regulatory environment in Europe, variations in standards of care, patient ascertainment and enrolment, a narrow therapeutic window and a lack of biomarkers of efficacy. In this context, SMA-Europe organized its first international workshop in July 2012 in Rome, gathering 34 scientists, clinicians and representatives of patient organizations to establish recommendations for improving clinical trials for SMAa. © 2013 Kayadjanian et al.; licensee BioMed Central Ltd.

Cite

CITATION STYLE

APA

Kayadjanian, N., Burghes, A., Finkel, R. S., Mercuri, E., Rouault, F., Schwersenz, I., & Talbot, K. (2013). SMA-Europe workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe. Orphanet Journal of Rare Diseases, 8(1). https://doi.org/10.1186/1750-1172-8-44

Register to see more suggestions

Mendeley helps you to discover research relevant for your work.

Already have an account?

Save time finding and organizing research with Mendeley

Sign up for free