Title |
SMA-EUROPE workshop report: opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe
|
---|---|
Published in |
Orphanet Journal of Rare Diseases, March 2013
|
DOI | 10.1186/1750-1172-8-44 |
Pubmed ID | |
Authors |
Nathalie Kayadjanian, Arthur Burghes, Richard S Finkel, Eugenio Mercuri, Francoise Rouault, Inge Schwersenz, Kevin Talbot |
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. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
France | 1 | 50% |
Italy | 1 | 50% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Science communicators (journalists, bloggers, editors) | 1 | 50% |
Scientists | 1 | 50% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Spain | 1 | 4% |
Unknown | 27 | 96% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Researcher | 6 | 21% |
Student > Master | 5 | 18% |
Student > Ph. D. Student | 5 | 18% |
Other | 2 | 7% |
Student > Doctoral Student | 2 | 7% |
Other | 3 | 11% |
Unknown | 5 | 18% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 6 | 21% |
Biochemistry, Genetics and Molecular Biology | 4 | 14% |
Agricultural and Biological Sciences | 3 | 11% |
Nursing and Health Professions | 3 | 11% |
Psychology | 2 | 7% |
Other | 5 | 18% |
Unknown | 5 | 18% |