Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA

Molecular Genetics and Metabolism Reports

Volumn 1, Issue 1, 2014, Pages 31-41

  Chinen, Yasutsugu ^a   Higa, Takeshi ^b   Tomatsu, Shunji ^c,d   Suzuki, Yasuyuki ^d   Orii, Tadao ^d   Hyakuna, Nobuyuki ^a  

^a UNIVERSITY OF THE RYUKYUS   (Japan)

^b Department of Pediatrics Okinawa Prefectural Nanbu Medical Center Children's Medical Center   (Japan)

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

^d GIFU UNIVERSITY   (Japan)

Author keywords

Bone marrow transplantation; Bone mineral density; GALNS; Mucopolysaccharidosis IVA; Respiratory function

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN; IMMUNOGLOBULIN ANTIBODY; METHOTREXATE; T LYMPHOCYTIC GLOBULIN ANTIBODY; UNCLASSIFIED DRUG; URONIC ACID; ERYTHROCYTE ANTIGEN; GLOBULIN; N ACETYLGALACTOSAMINE 6 SULFATASE; T LYMPHOCYTIC GLOBULIN;

ADOLESCENT; ALLOGENIC BONE MARROW TRANSPLANTATION; APNEA; ARTHRALGIA; ARTICLE; BODY WEIGHT; BONE DENSITY; CASE REPORT; DAILY LIFE ACTIVITY; DYSPNEA; ENZYME ACTIVITY; ENZYME ASSAY; GREATER TROCHANTER; HEARING IMPAIRMENT; HEPATOSPLENOMEGALY; HUMAN; LUNG FUNCTION; LYSOSOME STORAGE DISEASE; MALE; MORQUIO SYNDROME TYPE A; MUCOPOLYSACCHARIDOSIS; OPTIC DISK; SPONDYLOEPIPHYSEAL DYSPLASIA; VITAL CAPACITY; ACETABULUM; ANKLE FOOT ORTHOSIS; ATLANTO AXIAL FIXATION; ATLANTOAXIAL SUBLUXATION; BIRTH LENGTH; BIRTH WEIGHT; BODY HEIGHT; BODY MASS; BONE RADIOGRAPHY; BREATHING; CERVICAL FUSION; CERVICAL MYELOPATHY; CHROMOSOME ANALYSIS; COGNITION; CONTROLLED STUDY; DONOR; DUAL ENERGY X RAY ABSORPTIOMETRY; FEMUR EPIPHYSIS; FLUORESCENCE IN SITU HYBRIDIZATION; FORCED EXPIRATORY VOLUME; GLAUCOMA; HYPERMOBILE JOINT; IMMUNOSUPPRESSIVE TREATMENT; INTRAOCULAR PRESSURE; KARYOTYPE 46,XX; KYPHOSIS; LESSER TROCHANTER; LUMBAR SPINE; MORQUIO SYNDROME; MOVEMENT (PHYSIOLOGY); MUSCLE WEAKNESS; OCCIPITOFRONTAL CIRCUMFERENCE; OSTEOTOMY; PARAMETERS CONCERNING THE FETUS, NEWBORN AND PREGNANCY; PEAK EXPIRATORY FLOW; PLATYSPONDYLIA; POWERED WHEELCHAIR; RESPIRATORY STATUS; SLEEP; SNORING; SPINE FUSION; SPINE STABILIZATION; SPIROMETRY; STUDY; THORAX PAIN; ULNAR DEVIATION; VALGUS KNEE; VERTEBRAL CANAL STENOSIS; WORK;

EID: 84905184652     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2013.11.002     Document Type: Article

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