Literature DB >> 27118454

Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: the Dutch experience.

Diane F van Rappard1, Jaap J Boelens2, Martje E van Egmond3, Jurgen Kuball4, Peter M van Hasselt5, Kim J Oostrom6, Petra J W Pouwels7, Marjo S van der Knaap1, Carla E M Hollak8, Nicole I Wolf1.   

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Year:  2016        PMID： 27118454     DOI： 10.1182/blood-2016-03-708479

Source DB:  PubMed          Journal:  Blood        ISSN： 0006-4971            Impact factor:   22.113


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  18 in total

1.  Neurologic event-free survival demonstrates a benefit for SCID patients diagnosed by newborn screening.

Authors:  Christopher C Dvorak; Jennifer M Puck; Justin T Wahlstrom; Morna Dorsey; Alexis Melton; Morton J Cowan
Journal:  Blood Adv       Date:  2017-09-05

2.  Early and late outcomes after cord blood transplantation for pediatric patients with inherited leukodystrophies.

Authors:  Brigitte T A van den Broek; Kristin Page; Annalisa Paviglianiti; Janna Hol; Heather Allewelt; Fernanda Volt; Gerard Michel; Miguel Angel Diaz; Victoria Bordon; Tracey O'Brien; Peter J Shaw; Chantal Kenzey; Amal Al-Seraihy; Peter M van Hasselt; Andrew R Gennery; Eliane Gluckman; Vanderson Rocha; Annalisa Ruggeri; Joanne Kurtzberg; Jaap Jan Boelens
Journal:  Blood Adv       Date:  2018-01-04

3.  Demyelination load as predictor for disease progression in juvenile metachromatic leukodystrophy.

Authors:  Manuel Strölin; Ingeborg Krägeloh-Mann; Christiane Kehrer; Marko Wilke; Samuel Groeschel
Journal:  Ann Clin Transl Neurol       Date:  2017-05-16       Impact factor: 4.511

4.  Phenotypic variation between siblings with Metachromatic Leukodystrophy.

Authors:  Saskia Elgün; Jakob Waibel; Christiane Kehrer; Diane van Rappard; Judith Böhringer; Stefanie Beck-Wödl; Jennifer Just; Ludger Schöls; Nicole Wolf; Ingeborg Krägeloh-Mann; Samuel Groeschel
Journal:  Orphanet J Rare Dis       Date:  2019-06-11       Impact factor: 4.123

Review 5.  Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective.

Authors:  Shanice Beerepoot; Stefan Nierkens; Jaap Jan Boelens; Caroline Lindemans; Marianna Bugiani; Nicole I Wolf
Journal:  Orphanet J Rare Dis       Date:  2019-11-04       Impact factor: 4.123

Review 6.  CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges.

Authors:  Mariola J Edelmann; Gustavo H B Maegawa
Journal:  Front Mol Biosci       Date:  2020-11-12

7.  Allogenic hematopoietic stem cell transplantation in two siblings with adult metachromatic leukodystrophy and a systematic literature review.

Authors:  Cecilie Videbæk; Jette Stokholm; Henrik Sengeløv; Lone U Fjeldborg; Vibeke Andrée Larsen; Christian Krarup; Jørgen E Nielsen; Sabine Grønborg
Journal:  JIMD Rep       Date:  2021-05-06

Review 8.  Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.

Authors:  Rachele Penati; Francesca Fumagalli; Valeria Calbi; Maria Ester Bernardo; Alessandro Aiuti
Journal:  J Inherit Metab Dis       Date:  2017-05-30       Impact factor: 4.982

9.  Diffusion tensor imaging in metachromatic leukodystrophy.

Authors:  Diane F van Rappard; Marsh Königs; Marjan E Steenweg; Jaap Jan Boelens; Jaap Oosterlaan; Marjo S van der Knaap; Nicole I Wolf; Petra J W Pouwels
Journal:  J Neurol       Date:  2018-01-30       Impact factor: 4.849

10.  Metachromatic leukodystrophy and transplantation: remyelination, no cross-correction.

Authors:  Nicole I Wolf; Marjolein Breur; Bonnie Plug; Shanice Beerepoot; Aimee S R Westerveld; Diane F van Rappard; Sharon I de Vries; Maarten H P Kole; Adeline Vanderver; Marjo S van der Knaap; Caroline A Lindemans; Peter M van Hasselt; Jaap J Boelens; Ulrich Matzner; Volkmar Gieselmann; Marianna Bugiani
Journal:  Ann Clin Transl Neurol       Date:  2020-01-22       Impact factor: 4.511
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