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Molecular Genetics and Metabolism
|
February 7, 2015
A clinical approach to the diagnosis of patients with leukodystrophies and genetic leukoencephelopathies
Sumit Parikh, Geneviève Bernard, Richard J Leventer, et al.
Annals of the Rheumatic Diseases
|
June 8, 2014
Aicardi-Goutières syndrome harbours abundant systemic and brain-reactive autoantibodies
Eloy Cuadrado, Adeline Vanderver, Kristy J Brown, et al.
Human Mutation
|
March 4, 2020
Type II Alexander disease caused by splicing errors and aberrant overexpression of an uncharacterized GFAP isoform
Guy Helman, Asako Takanohashi, Tracy L Hagemann, et al.
Human Mutation
|
August 3, 2022
Type II Alexander disease caused by splicing errors and aberrant overexpression of an uncharacterized GFAP isoform
Guy Helman, Asako Takanohashi, Tracy L Hagemann, et al.
Pediatric Neurology
|
October 23, 2025
Critical Functional Domains in Pediatric Onset TUBB4A-Related Leukodystrophy: A Clinical and Caregiver's Perspective
Francesco Gavazzi, Virali Patel, Jacqueline A Erler, et al.
Journal of Inherited Metabolic Disease
|
October 23, 2023
Biochemical signatures of disease severity in multiple sulfatase deficiency
Laura A Adang, Samar Mowafy, Zackary M Herbst, et al.
Elife
|
May 29, 2020
<i>TUBB4A</i> mutations result in both glial and neuronal degeneration in an H-ABC leukodystrophy mouse model
Sunetra Sase, Akshata A Almad, C Alexander Boecker, et al.
American Journal of Human Genetics
|
August 23, 2011
Mutations of POLR3A encoding a catalytic subunit of RNA polymerase Pol III cause a recessive hypomyelinating leukodystrophy
Geneviève Bernard, Eliane Chouery, Maria Lisa Putorti, et al.
Biorxiv : the Preprint Server for Biology
|
April 1, 2024
Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations
Lucas Tricoli, Sunetra Sase, Julia Hacker, et al.
Molecular Genetics and Metabolism
|
February 5, 2018
Complex care of individuals with multiple sulfatase deficiency: Clinical cases and consensus statement
Rebecca Ahrens-Nicklas, Lars Schlotawa, Andrea Ballabio, et al.
Page
of 22
Search research articles
Search
Showing results (111-120 of 217) with videos related to
Sort By:
Page
of 22
Molecular Genetics and Metabolism
|
February 7, 2015
A clinical approach to the diagnosis of patients with leukodystrophies and genetic leukoencephelopathies
Sumit Parikh, Geneviève Bernard, Richard J Leventer, et al.
Annals of the Rheumatic Diseases
|
June 8, 2014
Aicardi-Goutières syndrome harbours abundant systemic and brain-reactive autoantibodies
Eloy Cuadrado, Adeline Vanderver, Kristy J Brown, et al.
Human Mutation
|
March 4, 2020
Type II Alexander disease caused by splicing errors and aberrant overexpression of an uncharacterized GFAP isoform
Guy Helman, Asako Takanohashi, Tracy L Hagemann, et al.
Human Mutation
|
August 3, 2022
Type II Alexander disease caused by splicing errors and aberrant overexpression of an uncharacterized GFAP isoform
Guy Helman, Asako Takanohashi, Tracy L Hagemann, et al.
Pediatric Neurology
|
October 23, 2025
Critical Functional Domains in Pediatric Onset TUBB4A-Related Leukodystrophy: A Clinical and Caregiver's Perspective
Francesco Gavazzi, Virali Patel, Jacqueline A Erler, et al.
Journal of Inherited Metabolic Disease
|
October 23, 2023
Biochemical signatures of disease severity in multiple sulfatase deficiency
Laura A Adang, Samar Mowafy, Zackary M Herbst, et al.
Elife
|
May 29, 2020
<i>TUBB4A</i> mutations result in both glial and neuronal degeneration in an H-ABC leukodystrophy mouse model
Sunetra Sase, Akshata A Almad, C Alexander Boecker, et al.
American Journal of Human Genetics
|
August 23, 2011
Mutations of POLR3A encoding a catalytic subunit of RNA polymerase Pol III cause a recessive hypomyelinating leukodystrophy
Geneviève Bernard, Eliane Chouery, Maria Lisa Putorti, et al.
Biorxiv : the Preprint Server for Biology
|
April 1, 2024
Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations
Lucas Tricoli, Sunetra Sase, Julia Hacker, et al.
Molecular Genetics and Metabolism
|
February 5, 2018
Complex care of individuals with multiple sulfatase deficiency: Clinical cases and consensus statement
Rebecca Ahrens-Nicklas, Lars Schlotawa, Andrea Ballabio, et al.
Page
of 22