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

Orphanet Journal of Rare Diseases

Volumn 8, Issue 1, 2013, Pages

  Kayadjanian, Nathalie ^a   Burghes, Arthur ^b   Finkel, Richard S ^c   Mercuri, Eugenio ^d   Rouault, Francoise ^e   Schwersenz, Inge ^f   Talbot, Kevin ^g  

^a TransBioMed Solutions LLC   (United States)

^b THE OHIO STATE UNIVERSITY COLLEGE OF MEDICINE   (United States)

^c NEMOURS CHILDREN S HOSPITAL   (United States)

^d CATHOLIC UNIVERSITY   (Italy)

^e Association Française Contre Les Myopathies   (France)

^f SMA EUROPE   (Germany)

^g UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

Clinical trials; Efficacy biomarkers; European regulations; Neonatal trials; Newborn screening; Patient registries; Phase III trial planning; Preclinical drug selection; Spinal muscular atrophy; Standards of care

Indexed keywords

BIOLOGICAL MARKER;

ARTICLE; CLINICAL TRIAL (TOPIC); DISEASE MARKER; ELECTROPHYSIOLOGY; EUROPE; HEALTH CARE COST; HUMAN; LIFESPAN; PATIENT CARE; SPINAL MUSCULAR ATROPHY; SURVIVAL;

CLINICAL TRIALS AS TOPIC; EUROPE; HUMANS; MUSCULAR ATROPHY, SPINAL;

EID: 84875079059     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/1750-1172-8-44     Document Type: Article

References (11)

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