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SMA-EUROPE workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe.
- Source :
-
Orphanet journal of rare diseases [Orphanet J Rare Dis] 2013 Mar 20; Vol. 8, pp. 44. Date of Electronic Publication: 2013 Mar 20. - Publication Year :
- 2013
-
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.
- Subjects :
- Europe
Humans
Clinical Trials as Topic
Muscular Atrophy, Spinal therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1750-1172
- Volume :
- 8
- Database :
- MEDLINE
- Journal :
- Orphanet journal of rare diseases
- Publication Type :
- Report
- Accession number :
- 23514578
- Full Text :
- https://doi.org/10.1186/1750-1172-8-44