Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

Orphanet Journal of Rare Diseases

Volumn 12, Issue 1, 2017, Pages

  Ghosh, Arunabha ^a,b   Shapiro, Elsa ^c,d   Rust, Stewart ^e   Delaney, Kathleen ^c   Parker, Samantha ^f   Shaywitz, Adam J ^g   Morte, Adelaida ^h   Bubb, Gillian ⁱ   Cleary, Maureen ^j   Bo, Tien ^k   Lavery, Christine ^l   Bigger, Brian W ^b   Jones, Simon A ^a  

^a UNIVERSITY OF MANCHESTER   (United Kingdom)

^b UNIVERSITY OF MANCHESTER   (United Kingdom)

^c Shapiro and Delaney LLC   (United States)

^d UNIVERSITY OF MINNESOTA   (United States)

^e ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^f LYSOGENE SAS   (France)

^g BIOMARIN PHARMACEUTICAL INC   (United States)

^h Esteve   (Spain)

ⁱ ALEXION PHARMACEUTICALS   (United States)

^j GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^k SHIRE   (United States)

^l MPS Society   (United Kingdom)

more..

Author keywords

Behaviour; Clinical trials; Cognitive assessment; Lysosomal storage disorders; mucopolysaccharidoses; Mucopolysaccharidosis Type III; Natural history; Quality of life; Sanfilippo syndrome

Indexed keywords

ADENOIDECTOMY; BAYLEY SCALES OF INFANT DEVELOPMENT; BEHAVIOR DISORDER; CLINICAL RESEARCH; COGNITION; COGNITIVE DEFECT; COHORT ANALYSIS; CONFERENCE PAPER; HUMAN; INTELLECTUAL IMPAIRMENT; LIMIT OF QUANTITATION; PATHOPHYSIOLOGY; POPULATION RESEARCH; PRACTICE GUIDELINE; PROSPECTIVE STUDY; SANFILIPPO SYNDROME; SLEEP DISORDER; TONSILLECTOMY; TREATMENT RESPONSE; CLINICAL TRIAL (TOPIC); ENZYME REPLACEMENT; ENZYMOLOGY; FEMALE; INFANT; LYSOSOME STORAGE DISEASE; MALE; METABOLISM; MUCOPOLYSACCHARIDOSIS; PHYSIOLOGY; PRESCHOOL CHILD; QUALITY OF LIFE;

CHILD, PRESCHOOL; CLINICAL TRIALS AS TOPIC; COGNITION; ENZYME REPLACEMENT THERAPY; FEMALE; HUMANS; INFANT; LYSOSOMAL STORAGE DISEASES; MALE; MUCOPOLYSACCHARIDOSES; MUCOPOLYSACCHARIDOSIS III; QUALITY OF LIFE;

EID: 85021311851     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-017-0675-4     Document Type: Conference Paper

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