2007
Buts thérapeutiques dans la maladie de Gaucher Therapeutic objectives in Gaucher disease
Mistry P, Germain D. Buts thérapeutiques dans la maladie de Gaucher Therapeutic objectives in Gaucher disease. La Revue De Médecine Interne 2007, 28: 171-175. PMID: 18228683, DOI: 10.1016/s0248-8663(07)78876-8.Peer-Reviewed Original ResearchConceptsGaucher diseaseLife-threatening complicationsStandard of careMaximal clinical benefitRemarkable safety profileTreatment of GDQuality of lifeType 1 GDBone crisesLifelong treatmentClinical benefitSafety profileAsymptomatic phaseTherapeutic goalsDisease evolutionTherapeutic objectivesCareful monitoringLa maladieSkeletal damageNorth American expertsDiseaseDisease phenotypePatientsTreatmentOptimal time
2006
Therapeutic goals in Gaucher disease
Mistry P, Germain DP. Therapeutic goals in Gaucher disease. La Revue De Médecine Interne 2006, 27: s30-s33. PMID: 16644400, DOI: 10.1016/s0248-8663(06)80009-3.Peer-Reviewed Original ResearchConceptsGaucher diseaseTherapeutic goalsType 1 Gaucher diseaseLong-life treatmentLife-threatening complicationsStandard of careRemarkable safety profileQuality of lifeBone crisesPrevent complicationsSafety profileAsymptomatic phaseDisease evolutionCareful monitoringDiseaseSkeletal damageComplicationsPatientsGaucher phenotypeTreatmentOptimal timeTolerabilityAmerican expertsSeverityCare
2004
Therapeutic goals in the treatment of Gaucher disease
Pastores GM, Weinreb NJ, Aerts H, Andria G, Cox TM, Giralt M, Grabowski GA, Mistry PK, Tylki-Szymańska A. Therapeutic goals in the treatment of Gaucher disease. Seminars In Hematology 2004, 41: 4-14. PMID: 15468045, DOI: 10.1053/j.seminhematol.2004.07.009.Peer-Reviewed Original ResearchMeSH KeywordsEnzyme TherapyEvidence-Based MedicineGaucher DiseaseHumansPractice Guidelines as TopicQuality of LifeTreatment OutcomeConceptsEnzyme replacement therapyGaucher diseaseTherapeutic responseTherapeutic goalsNon-neuronopathic (type 1) Gaucher diseaseType 1 Gaucher diseaseVariable disease patternGoal of treatmentCommon lysosomal storage disorderEvidence-based consensusExtensive clinical experienceQuality of lifeImplementation of treatmentLysosomal storage disorderPulmonary diseaseReplacement therapyMultisystem conditionIndividualized managementIndividual patientsClinical experienceDisease patternsGrowth retardationDiseaseStorage disorderSkeletal pathology