Literature DB >> 34212263

New disease modifying therapies for two genetic childhood-onset neurometabolic disorders (metachromatic leucodystrophy and adrenoleucodystrophy).

Antonio Federico1, Marianne de Visser2.   

Abstract

Entities:  

Year:  2021        PMID: 34212263     DOI: 10.1007/s10072-021-05412-x

Source DB:  PubMed          Journal:  Neurol Sci        ISSN: 1590-1874            Impact factor:   3.307


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  3 in total

1.  Brain cell type-specific endocytosis of arylsulfatase A identifies limitations of enzyme-based therapies for metachromatic leukodystrophy.

Authors:  Debora Kaminski; Claudia Yaghootfam; Frank Matthes; Annika Reßing; Volkmar Gieselmann; Ulrich Matzner
Journal:  Hum Mol Genet       Date:  2021-02-04       Impact factor: 6.150

2.  Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

Authors:  Florian Eichler; Christine Duncan; Patricia L Musolino; Paul J Orchard; Satiro De Oliveira; Adrian J Thrasher; Myriam Armant; Colleen Dansereau; Troy C Lund; Weston P Miller; Gerald V Raymond; Raman Sankar; Ami J Shah; Caroline Sevin; H Bobby Gaspar; Paul Gissen; Hernan Amartino; Drago Bratkovic; Nicholas J C Smith; Asif M Paker; Esther Shamir; Tara O'Meara; David Davidson; Patrick Aubourg; David A Williams
Journal:  N Engl J Med       Date:  2017-10-04       Impact factor: 91.245

3.  Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice.

Authors:  Emilie Audouard; Valentin Oger; Béatrix Meha; Nathalie Cartier; Caroline Sevin; Françoise Piguet
Journal:  Front Mol Neurosci       Date:  2021-05-21       Impact factor: 5.639

  3 in total
  2 in total

1.  Indications for haematopoietic cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2022.

Authors:  John A Snowden; Isabel Sánchez-Ortega; Selim Corbacioglu; Grzegorz W Basak; Christian Chabannon; Rafael de la Camara; Harry Dolstra; Rafael F Duarte; Bertram Glass; Raffaella Greco; Arjan C Lankester; Mohamad Mohty; Bénédicte Neven; Régis Peffault de Latour; Paolo Pedrazzoli; Zinaida Peric; Ibrahim Yakoub-Agha; Anna Sureda; Nicolaus Kröger
Journal:  Bone Marrow Transplant       Date:  2022-05-19       Impact factor: 5.174

Review 2.  Pediatric Adrenal Insufficiency: Challenges and Solutions.

Authors:  Daniela Nisticò; Benedetta Bossini; Simone Benvenuto; Maria Chiara Pellegrin; Gianluca Tornese
Journal:  Ther Clin Risk Manag       Date:  2022-01-11       Impact factor: 2.423

  2 in total

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