Literature DB >> 29680197

Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.

Pramod K Mistry1, Manisha Balwani2, Hagit N Baris3, Hadhami Ben Turkia4, T Andrew Burrow5, Joel Charrow6, Gerald F Cox7, Sumita Danda8, Marta Dragosky9, Guillermo Drelichman10, Amal El-Beshlawy11, Cristina Fraga12, Selena Freisens13, Sebastiaan Gaemers13, Evgueniy Hadjiev14, Priya S Kishnani15, Elena Lukina16, Pierre Maison-Blanche17, Ana Maria Martins18, Gregory Pastores19, Milan Petakov20, M Judith Peterschmitt13, Hanna Rosenbaum21, Barry Rosenbloom22, Lisa H Underhill13, Timothy M Cox23.   

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Year:  2018        PMID： 29680197     DOI： 10.1016/j.bcmd.2018.04.001

Source DB:  PubMed          Journal:  Blood Cells Mol Dis        ISSN： 1079-9796            Impact factor:   3.039


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  7 in total

1.  Stereoselective Synthesis of C-2 Alkylated Trihydroxypiperidines: Novel Pharmacological Chaperones for Gaucher Disease.

Authors:  Francesca Clemente; Camilla Matassini; Andrea Goti; Amelia Morrone; Paolo Paoli; Francesca Cardona
Journal:  ACS Med Chem Lett       Date:  2019-02-08       Impact factor: 4.345

2.  Lysosomal Diseases and Neuropsychiatry: Opportunities to Rebalance the Mind.

Authors:  Timothy M Cox
Journal:  Front Mol Biosci       Date:  2020-08-26

Review 3.  Diagnosing neuronopathic Gaucher disease: New considerations and challenges in assigning Gaucher phenotypes.

Authors:  Emily C Daykin; Emory Ryan; Ellen Sidransky
Journal:  Mol Genet Metab       Date:  2021-01-09       Impact factor: 4.797

4.  Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.

Authors:  M Judith Peterschmitt; Selena Freisens; Lisa H Underhill; Meredith C Foster; Grace Lewis; Sebastiaan J M Gaemers
Journal:  Orphanet J Rare Dis       Date:  2019-06-07       Impact factor: 4.123

5.  HEPES-buffering of bicarbonate-containing culture medium perturbs lysosomal glucocerebrosidase activity.

Authors:  Martijn J C van der Lienden; Jan Aten; Rolf G Boot; Marco van Eijk; Johannes M F G Aerts; Chi-Lin Kuo
Journal:  J Cell Biochem       Date:  2022-03-21       Impact factor: 4.480

Review 6.  Glucocerebrosidase: Functions in and Beyond the Lysosome.

Authors:  Daphne E C Boer; Jeroen van Smeden; Joke A Bouwstra; Johannes M F G Aerts
Journal:  J Clin Med       Date:  2020-03-09       Impact factor: 4.241

Review 7.  Glycosphingolipids and Infection. Potential New Therapeutic Avenues.

Authors:  Johannes M F G Aerts; M Artola; M van Eijk; M J Ferraz; R G Boot
Journal:  Front Cell Dev Biol       Date:  2019-12-06
  7 in total

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