TY - JOUR
T1 - SMA-EUROPE workshop report: Opportunities and challenges in developing clinical trials for spinal muscular atrophy in Europe
AU - Kayadjanian, Nathalie
AU - Burghes, Arthur
AU - Finkel, Richard S
AU - Mercuri, Eugenio Maria
AU - Rouault, Francoise
AU - Schwersenz, Inge
AU - Talbot, Kevin
PY - 2013
Y1 - 2013
N2 - 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.
AB - 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.
KW - Clinical Trials as Topic
KW - Europe
KW - Humans
KW - Muscular Atrophy, Spinal
KW - Clinical Trials as Topic
KW - Europe
KW - Humans
KW - Muscular Atrophy, Spinal
UR - http://hdl.handle.net/10807/53552
U2 - 10.1186/1750-1172-8-44
DO - 10.1186/1750-1172-8-44
M3 - Article
SN - 1750-1172
VL - 8
SP - 44
EP - 44
JO - Orphanet Journal of Rare Diseases
JF - Orphanet Journal of Rare Diseases
ER -