Therapies of mucopolysaccharidosis IVA (Morquio A syndrome)

Expert Opinion on Orphan Drugs

Volumn 1, Issue 10, 2013, Pages 805-818

  Tomatsu, Shunji ^a   Alméciga Díaz, Carlos J ^b   Barbosa, Hector ^b   Montaño, Adriana M ^c   Barrera, Luis A ^b   Shimada, Tsutomu ^a   Yasuda, Eriko ^a   Mackenzie, William G ^a   Mason, Robert W ^a   Suzuki, Yasuyuki ^d   Orii, Kenji E ^d   Orii, Tadao ^d  

^a NEMOURS ALFRED I DUPONT HOSPITAL FOR CHILDREN   (United States)

^b PONTIFICIA UNIVERSIDAD JAVERIANA   (Colombia)

^c SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d GIFU UNIVERSITY   (Japan)

Author keywords

Enzyme replacement therapy; GALNS; Gene therapy; Keratan sulfate; Mucopolysaccharidosis IVA; Tandem mass spectrometry

Indexed keywords

BETA GLUCURONIDASE; BETA N ACETYLHEXOSAMINIDASE; HYDROXYAPATITE; LENTIVIRUS VECTOR; MANNITOL; MINICIRCLE DNA; OSTEOCLAST DIFFERENTIATION FACTOR; PLACEBO; RETROVIRUS VECTOR; TUMOR NECROSIS FACTOR;

ADENO ASSOCIATED VIRUS; ARTHRALGIA; BONE DYSPLASIA; BONE LESION; CARTILAGE CELL; CLINICAL TRIAL (TOPIC); COMPUTER ASSISTED TOMOGRAPHY; ENZYME ACTIVITY; ENZYME REPLACEMENT; GENE THERAPY; HALF LIFE TIME; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; HYPOPHOSPHATASIA; IMMUNOMODULATION; JOINT STIFFNESS; MAROTEAUX LAMY SYNDROME; MORQUIO A SYNDROME; MORQUIO SYNDROME; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; NONVIRAL GENE THERAPY; OSTEOARTHRITIS; REVIEW; SANFILIPPO SYNDROME; VIRAL GENE THERAPY;

EID: 84896735986     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2013.846853     Document Type: Review

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