Clinical trials in rare disease: Challenges and opportunities

Journal of Child Neurology

Volumn 28, Issue 9, 2013, Pages 1142-1150

  Augustine, Erika F ^a   Adams, Heather R ^a   Mink, Jonathan W ^a  

^a UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

Author keywords

Batten disease; clinical trials; lysosomal storage disease; neuronal ceroid lipofuscinosis; rare disease

Indexed keywords

ACETYLCYSTEINE; ALPHA TOCOPHEROL; AMANTADINE; ASCORBIC ACID; BENSERAZIDE; BUTYLCRESOL; CITALOPRAM; FENTANYL; FLUPIRTINE; GLUTATHIONE PEROXIDASE; LAMOTRIGINE; LEVODOPA; MELATONIN; MERCAPTAMINE; METHIONINE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; OLANZAPINE; ORPHENADRINE; POLYUNSATURATED FATTY ACID; PREDNISOLONE; PYRIDOXINE; RIBOFLAVIN; RISPERIDONE; SELEGILINE; SELENIUM; SODIUM SELENITE;

ARTICLE; BLOOD BRAIN BARRIER; BONE MARROW TRANSPLANTATION; CENTRAL NERVOUS SYSTEM; CHILDHOOD DISEASE; ENZYME ACTIVITY; FUNDING; GENE TRANSFER; HEALTH CARE ORGANIZATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; METHODOLOGY; NEURONAL CEROID LIPOFUSCINOSIS; PALLIDOTOMY; PATHOPHYSIOLOGY; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); RARE DISEASE; TREATMENT OUTCOME; VITAMIN BLOOD LEVEL;

BATTEN DISEASE; CLINICAL TRIALS; LYSOSOMAL STORAGE DISEASE; NEURONAL CEROID LIPOFUSCINOSIS; RARE DISEASE;

CLINICAL TRIALS AS TOPIC; HUMANS; NEURONAL CEROID-LIPOFUSCINOSES; RARE DISEASES; RESEARCH DESIGN;

EID: 84883688333     PISSN: 08830738     EISSN: 17088283     Source Type: Journal    
DOI: 10.1177/0883073813495959     Document Type: Article

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