Literature DB >> 19308038

Pathological evidence of Wolman's disease following hematopoietic stem cell transplantation despite correction of lysosomal acid lipase activity.

M M Gramatges, C C Dvorak, D P Regula, G M Enns, K Weinberg, R Agarwal.   

Abstract

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Year:  2009        PMID： 19308038     DOI： 10.1038/bmt.2009.57

Source DB:  PubMed          Journal:  Bone Marrow Transplant        ISSN： 0268-3369            Impact factor:   5.483


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  10 in total

Review 1.  The role of sebelipase alfa in the treatment of lysosomal acid lipase deficiency.

Authors:  Angelika L Erwin
Journal:  Therap Adv Gastroenterol       Date:  2017-04-26       Impact factor: 4.409

Review 2.  Early steps in steroidogenesis: intracellular cholesterol trafficking.

Authors:  Walter L Miller; Himangshu S Bose
Journal:  J Lipid Res       Date:  2011-10-05       Impact factor: 5.922

Review 3.  Lysosomal lipid storage diseases.

Authors:  Heike Schulze; Konrad Sandhoff
Journal:  Cold Spring Harb Perspect Biol       Date:  2011-06-01       Impact factor: 10.005

4.  Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease.

Authors:  Jane E Potter; Gemma Petts; Arunabha Ghosh; Fiona J White; Jane L Kinsella; Stephen Hughes; Jane Roberts; Adam Hodgkinson; Kathryn Brammeier; Heather Church; Christine Merrigan; Joanne Hughes; Pamela Evans; Helen Campbell; Denise Bonney; William G Newman; Brian W Bigger; Alexander Broomfield; Simon A Jones; Robert F Wynn
Journal:  Orphanet J Rare Dis       Date:  2021-05-21       Impact factor: 4.123

Review 5.  Novel treatment options for lysosomal acid lipase deficiency: critical appraisal of sebelipase alfa.

Authors:  Kim Su; Emma Donaldson; Reena Sharma
Journal:  Appl Clin Genet       Date:  2016-10-17

6.  Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study.

Authors:  Simon A Jones; Sandra Rojas-Caro; Anthony G Quinn; Mark Friedman; Sachin Marulkar; Fatih Ezgu; Osama Zaki; J Jay Gargus; Joanne Hughes; Dominique Plantaz; Roshni Vara; Stephen Eckert; Jean-Baptiste Arnoux; Anais Brassier; Kim-Hanh Le Quan Sang; Vassili Valayannopoulos
Journal:  Orphanet J Rare Dis       Date:  2017-02-08       Impact factor: 4.123

Review 7.  Targeting Wolman Disease and Cholesteryl Ester Storage Disease: Disease Pathogenesis and Therapeutic Development.

Authors:  Francis Aguisanda; Natasha Thorne; Wei Zheng
Journal:  Curr Chem Genom Transl Med       Date:  2017-01-30

Review 8.  Precision Medicine for Lysosomal Disorders.

Authors:  Filippo Pinto E Vairo; Diana Rojas Málaga; Francyne Kubaski; Carolina Fischinger Moura de Souza; Fabiano de Oliveira Poswar; Guilherme Baldo; Roberto Giugliani
Journal:  Biomolecules       Date:  2020-07-26

9.  Sebelipase alfa enzyme replacement therapy in Wolman disease: a nationwide cohort with up to ten years of follow-up.

Authors:  Tanguy Demaret; Florence Lacaille; Camille Wicker; Jean-Baptiste Arnoux; Juliette Bouchereau; Claire Belloche; Cyril Gitiaux; David Grevent; Christine Broissand; Dalila Adjaoud; Marie-Thérèse Abi Warde; Dominique Plantaz; Soumeya Bekri; Pascale de Lonlay; Anaïs Brassier
Journal:  Orphanet J Rare Dis       Date:  2021-12-14       Impact factor: 4.123

10.  Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants.

Authors:  Simon A Jones; Vassili Valayannopoulos; Eugene Schneider; Stephen Eckert; Maryam Banikazemi; Martin Bialer; Stephen Cederbaum; Alicia Chan; Anil Dhawan; Maja Di Rocco; Jennifer Domm; Gregory M Enns; David Finegold; J Jay Gargus; Ornella Guardamagna; Christian Hendriksz; Iman G Mahmoud; Julian Raiman; Laila A Selim; Chester B Whitley; Osama Zaki; Anthony G Quinn
Journal:  Genet Med       Date:  2015-08-27       Impact factor: 8.822
  10 in total

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