Literature DB >> 26096743

The history and accomplishments of the ICGG Gaucher registry.

Neal J Weinreb1, Paige Kaplan2.   

Abstract

Mesh：

Year:  2015        PMID： 26096743     DOI： 10.1002/ajh.24054

Source DB:  PubMed          Journal:  Am J Hematol        ISSN： 0361-8609            Impact factor:   10.047


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  7 in total

1.  MGUS, lymphoplasmacytic malignancies, and Gaucher disease: the significance of the clinical association.

Authors:  Neal J Weinreb; Pramod K Mistry; Barry E Rosenbloom; Madhav V Dhodapkar
Journal:  Blood       Date:  2018-04-12       Impact factor: 22.113

2.  Design of a framework for the deployment of collaborative independent rare disease-centric registries: Gaucher disease registry model.

Authors:  Matthew I Bellgard; Kathryn R Napier; Alan H Bittles; Jeffrey Szer; Sue Fletcher; Nikolajs Zeps; Adam A Hunter; Jack Goldblatt
Journal:  Blood Cells Mol Dis       Date:  2017-01-27       Impact factor: 3.039

Review 3.  Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry.

Authors:  Joseph Muenzer; Simon A Jones; Anna Tylki-Szymańska; Paul Harmatz; Nancy J Mendelsohn; Nathalie Guffon; Roberto Giugliani; Barbara K Burton; Maurizio Scarpa; Michael Beck; Yvonne Jangelind; Elizabeth Hernberg-Stahl; Maria Paabøl Larsen; Tom Pulles; David A H Whiteman
Journal:  Orphanet J Rare Dis       Date:  2017-05-02       Impact factor: 4.123

4.  A Quantitative Systems Pharmacology Model of Gaucher Disease Type 1 Provides Mechanistic Insight Into the Response to Substrate Reduction Therapy With Eliglustat.

Authors:  Ruth Abrams; Chanchala D Kaddi; Mengdi Tao; Randolph J Leiser; Giulia Simoni; Federico Reali; John Tolsma; Paul Jasper; Zachary van Rijn; Jing Li; Bradley Niesner; Jeffrey S Barrett; Luca Marchetti; M Judith Peterschmitt; Karim Azer; Susana Neves-Zaph
Journal:  CPT Pharmacometrics Syst Pharmacol       Date:  2020-06-19

5.  The International Collaborative Gaucher Group GRAF (Gaucher Risk Assessment for Fracture) score: a composite risk score for assessing adult fracture risk in imiglucerase-treated Gaucher disease type 1 patients.

Authors:  Patrick Deegan; Aneal Khan; José Simon Camelo; Julie L Batista; Neal Weinreb
Journal:  Orphanet J Rare Dis       Date:  2021-02-18       Impact factor: 4.123

6.  The Italian neuromuscular registry: a coordinated platform where patient organizations and clinicians collaborate for data collection and multiple usage.

Authors:  Anna Ambrosini; Daniela Calabrese; Francesco Maria Avato; Felice Catania; Guido Cavaletti; Maria Carmela Pera; Antonio Toscano; Giuseppe Vita; Lucia Monaco; Davide Pareyson
Journal:  Orphanet J Rare Dis       Date:  2018-10-04       Impact factor: 4.123

7.  Rare disease registries: potential applications towards impact on development of new drug treatments.

Authors:  Marijke C Jansen-van der Weide; Charlotte M W Gaasterland; Kit C B Roes; Caridad Pontes; Roser Vives; Arantxa Sancho; Stavros Nikolakopoulos; Eric Vermeulen; Johanna H van der Lee
Journal:  Orphanet J Rare Dis       Date:  2018-09-05       Impact factor: 4.123
  7 in total

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