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February 5, 2019

− 当社の希少疾患への取り組みを示す12件のプレゼンテーションには3件のライソゾーム病が含まれる

当社は、このたび、2019年2月4日から8日までフロリダ州オーランドで開催される第15回WORLDSymposium年次総会で、11件のポスタープレゼンテーションと1件のオーラルプレゼンテーションを含む12件の発表を行いますのでお知らせします。プレゼンテーションは、ハンター症候群(ムコ多糖症II型あるいはMPS IIと言われている)、I型ゴーシェ病、ファブリー病および異染性白質ジストロフィー(MLD)を含むライソゾーム病(LSD)に関する研究開発データで構成されています。







会場:ET Regency Ballroom U

Intrathecally administered recombinant human arylsulfatase A in patients with late-infantile metachromatic leukodystrophy: Phase 2b clinical trial design



会場:ET Regency Ballroom R

#210 Clinical characteristics of patients with neuronopathic and non-neuronopathic Mucopolysaccharidosis type II: Data from the Hunter Outcome Survey (HOS)



#101 Gaucher disease (GD)-specific patient-reported outcome (PRO) measures for clinical monitoring and for clinical trials



#209 Long-term analysis of velaglucerase alfa-treated patients with Gaucher disease who entered the Gaucher Outcome Survey real-life registry (GOS)



会場:ET Regency Ballroom R

#246 Characteristics of patients with Mucopolysaccharidosis type II who have received a bone marrow transplant: data from the Hunter Outcome Survey (HOS)



#245 Evaluation of the long-term treatment effects of idursulfase using statistical modelling: Data from the Hunter Outcome Survey (HOS)



#244 Neurodevelopmental status and adaptive behaviour of pediatric patients with Hunter syndrome: A longitudinal observational study



#387 Analysis of cognitive ability and adaptive behaviour assessment tools used in an observational study of patients with Hunter syndrome



#316 Study to determine predictive potential of an algorithm for earlier diagnosis of Gaucher disease: Retrospective biobank study utilizing real-world data available in Finland



#LB-34  A charitable access program for underserved lysosomal disease patients worldwide



#LB-53  Renoprotective effect of agalsidase alfa: 12-year follow-up of male Fabry patients



#LB-20  Classification of genetic variants in patients with Fabry disease enrolled in the Fabry Outcome Survey (FOS)