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1 . Academic Journal
The STAGED-PKD 2-Stage Adaptive Study With a Patient Enrichment Strategy and Treatment Effect Modeling for Improved Study Design Efficiency in Patients With ADPKDPlain-Language Summary
저자
by
Ronald D. Perrone
;
Ali Hariri
;
Pascal Minini
;
Curie Ahn
;
Arlene B. Chapman
, et al.
소스
Kidney Medicine, Vol 4, Iss 10, Pp 100538- (2022)
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7 . Academic Journal
Economic Burden of Hepatitis in South Korea
저자
by
Hugh Watson
;
Jeffrey Encinas
;
Pascal Minini
.
소스
Japanese Journal of Infectious Diseases. 2016, 69(4):354
Open Access (JSTAGE)
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5 .
Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial
저자
by
Raphael Schiffmann
;
Timothy M Cox
;
Jean-François Dedieu
;
Sebastiaan J M Gaemers
;
Julia B Hennermann
, et al.
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Open Access (OpenAIRE)
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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
Web of Science
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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 .
Venglustat, a novel brain-penetrant glucosylceramide synthase inhibitor, for GM2 gangliosidosis and related diseases: Phase 3 AMETHIST trial design
저자
by
Miloš Petrović
;
Riliang Zheng
;
Carla Canabarro
;
Pascal Minini
;
Isabela Batsu
.
소스
Molecular Genetics and Metabolism
. 135:S96-S97
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7 .
A study to evaluate the effect of venglustat on neuropathic and abdominal pain in symptomatic adult patients with Fabry disease
저자
by
Robert Hopkin
;
Joseph Kupferman
;
Patrick B. Deegan
;
Pascal Minini
;
Harmonie Goyeau
, et al.
소스
Molecular Genetics and Metabolism
. 135:S56-S57
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