Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: The ZAGAL project

Haematologica

Volumn 94, Issue 12, 2009, Pages 1771-1775

  Giraldo, Pilar ^a,b,h,i   Alfonso, Pilar ^b,i   Atutxa, Koldo ^c   Fernández Galán, María A ^c   Barez, Abelardo ^d   Franco, Rafael ^e   Alonso, Dora ^f   Martin, Alejandro ^g   Latre, Paz ^h   Pocovi, Miguel ^a,b,h,i  

^a HOSPITAL UNIVERSITARIO MIGUEL SERVET   (Spain)

^b CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED DE ENFERMEDADES RARAS CIBERER   (Spain)

^c Haematology Department   (Spain)

^d HOSPITAL NUESTRA SEÑORA DE SONSOLES   (Spain)

^e Haematology Department   (Spain)

^f HOSPITAL UNIVERSITARIO VIRGEN DEL ROCÍO   (Spain)

^g HOSPITAL VIRGEN DE LA CONCHA   (Spain)

^h Virgen de la Concha Hospital ^*  (Spain)

ⁱ INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD   (Spain)

Author keywords

Efficacy and safety; Maintenance; Real clinical setting; Substrate reduction therapy; Type 1 Gaucher disease

Indexed keywords

BIOLOGICAL MARKER; CHITOTRIOSIDASE; HEMOGLOBIN; MIGLUSTAT; PROTEIN PARC;

ADOLESCENT; ADULT; ARTICLE; BONE MARROW; CELL INFILTRATION; CLINICAL ARTICLE; CLINICAL PRACTICE; CLINICAL TRIAL; DISEASE SEVERITY; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; ENZYME REPLACEMENT; FEMALE; GASTROINTESTINAL SYMPTOM; GAUCHER DISEASE; HEMOGLOBIN BLOOD LEVEL; HUMAN; LIVER WEIGHT; LONG TERM CARE; LOW CARBOHYDRATE DIET; MAINTENANCE THERAPY; MALE; OPEN STUDY; OUTPATIENT DEPARTMENT; PROSPECTIVE STUDY; QUALITY OF LIFE; SPLEEN SIZE; THROMBOCYTE COUNT; TREATMENT DURATION; WEIGHT GAIN; WEIGHT REDUCTION;

1-DEOXYNOJIRIMYCIN; ADOLESCENT; ADULT; ALPHA-GLUCOSIDASES; CHEMOKINES, CC; CHILD; ENZYME INHIBITORS; FEMALE; GAUCHER DISEASE; HEXOSAMINIDASES; HUMANS; LIVER; MALE; MIDDLE AGED; ORGAN SIZE; PROSPECTIVE STUDIES; QUALITY OF LIFE; QUESTIONNAIRES; SPLEEN; TIME FACTORS; TREATMENT OUTCOME; WEIGHT LOSS; YOUNG ADULT;

EID: 73049101383     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2009.008078     Document Type: Article

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