title: SMA-EUROPE workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe.
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.
authors.forename: Nathalie
authors.initials: N
authors.lastname: Kayadjanian
authors.forename: Arthur
authors.initials: A
authors.lastname: Burghes
authors.forename: Richard S
authors.initials: RS
authors.lastname: Finkel
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
authors.forename: Francoise
authors.initials: F
authors.lastname: Rouault
authors.forename: Inge
authors.initials: I
authors.lastname: Schwersenz
authors.forename: Kevin
authors.initials: K
authors.lastname: Talbot
mesh_terms: D002986:Clinical Trials as Topic; D005060:Europe; D006801:Humans; D009134:Muscular Atrophy, Spinal / Q000628:therapy*