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1 . Academic Journal
The diagnostic journey for patients with late-onset GM2 Gangliosidoses
저자
by
Mariah C. Lopshire
;
Cynthia Tifft
;
John Burns
;
Rebecca Gould
;
Riliang Zheng
, et al.
소스
Molecular Genetics and Metabolism Reports, Vol 37, Iss , Pp 101014- (2023)
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7 . Academic Journal
Treatment adherence and effect of concurrent statin intensity on the efficacy and safety of alirocumab in a real-life setting: results from ODYSSEY APPRISE
저자
by
Maciej Banach
;
José Luis López-Sendon
;
Maurizio Averna
;
Bertrand Cariou
;
Megan Loy
, et al.
소스
Archives of Medical Science, Vol 18, Iss 2, Pp 285-292 (2021)
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7 .
AMETHIST: Baseline characteristics from a phase 3 trial of venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, in GM2-gangliosidoses and related diseases
저자
by
Cynthia J. Tifft
;
Isabela Batsu
;
Riliang Zheng
;
Pascal Minini
.
소스
Molecular Genetics and Metabolism
. 138:107335
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6 .
Changes in hematologic and visceral manifestations over time following imiglucerase initiation in Gaucher disease type 1 and type 3 pediatric patients in the ICGG Gaucher Registry
저자
by
Pramod K. Mistry
;
Pablo Bianculli
;
Isabela Batsu
;
Walter Heine
;
Antonio Oliveira-dos-Santos
, et al.
소스
Molecular Genetics and Metabolism
. 138:107225
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7 .
Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for Gaucher disease type 3: Phase 3 LEAP2MONO trial design
저자
by
Walter Heine
;
Isabela Batsu
;
Pascal Minini
;
Dmitry Cherkasov
.
소스
Molecular Genetics and Metabolism
. 138:107144
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6 .
Fine-motor functioning and its relevance to activities of daily living in adults with late-onset GM2 gangliosidosis: Analysis of baseline data of AMETHIST trial
저자
by
Timothy M. Cox
;
Maria B. Rodriguez
;
Valérie Dyevre
;
Jerome Msihid
;
Chad Gwaltney
, et al.
소스
Molecular Genetics and Metabolism
. 138:107063
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7 .
Two years of venglustat combined with imiglucerase shows continued positive effects on neurological features and brain connectivity in adults with Gaucher disease type 3
저자
by
Isabela Batsu
;
Riliang Zheng
;
Pascal Minini
;
Dmitry Cherkasov
;
David Scott
, et al.
소스
Molecular Genetics and Metabolism
. 138:107024
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7 .
ELIKIDS: Baseline characteristics from the eliglustat substrate reduction therapy trial in children with Gaucher disease type 1 or type 3
저자
by
Pilar Giraldo
;
Gulden Gokcay
;
Isabela Batsu
;
Julie Kissell
;
Maria G. Perichon
, et al.
소스
Molecular Genetics and Metabolism
. 138:107113
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7 .
Gaucher disease type 3 patient journey: Results from interviews with neuronopathic Gaucher disease patients highlight the challenges of diagnosis and care
저자
by
Laith Refaei
;
Davorka Sekulic
;
Andrew Doerr
;
Julia McCrone
;
Rebecca Gould
, et al.
소스
Molecular Genetics and Metabolism
. 138:107286
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7 .
Safety and efficacy of alirocumab in a real-life setting: the ODYSSEY APPRISE study
저자
by
Jose Lopez-Sendon
;
Maurizio Averna
;
Daniel Gaudet
;
Alexia Letierce
;
Maciej Banach
, et al.
소스
European Journal of Preventive Cardiology
. 28:1864-1872
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