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European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society
|
January 23, 2017
Late onset Krabbe disease due to the new GALC p.Ala543Pro mutation, with intriguingly high residual GALC activity in vitro
Inge Krägeloh-Mann, Klaus Harzer, Kevin Rostásy, et al.
Human Mutation
|
December 26, 2001
NPC1: Complete genomic sequence, mutation analysis, and characterization of haplotypes
Peter Bauer, Rupert Knoblich, Claudia Bauer, et al.
JIMD Reports
|
March 17, 2021
Long-term disease course of two patients with multiple sulfatase deficiency differs from metachromatic leukodystrophy in a broad cohort
Stefanie Beck-Wödl, Christiane Kehrer, Klaus Harzer, et al.
JIMD Reports
|
July 13, 2022
Extremely low arylsulfatase A enzyme activity does not necessarily cause symptoms: A long-term follow-up and review of the literature
Lucia Laugwitz, Vidiyaah Santhanakumaran, Mareike Spieker, et al.
Molecular Genetics and Metabolism
|
February 5, 2023
Corrigendum to "Predicting clinical phenotypes of metachromatic leukodystrophy based on the arylsulfatase A activity and the ARSA genotype? - Chances and challenges" Mol Genet Metab/Vol 137/Issue 3/2022/ 273-282
Vidiyaah Santhanakumaran, Samuel Groeschel, Klaus Harzer, et al.
Molecular Genetics and Metabolism
|
October 14, 2022
Predicting clinical phenotypes of metachromatic leukodystrophy based on the arylsulfatase A activity and the ARSA genotype? - Chances and challenges
Vidiyaah Santhanakumaran, Samuel Groeschel, Klaus Harzer, et al.
Human Mutation
|
August 2, 2017
Enzymatic characterization of novel arylsulfatase A variants using human arylsulfatase A-deficient immortalized mesenchymal stromal cells
Judith Böhringer, René Santer, Neele Schumacher, et al.
Lancet (London, England)
|
November 22, 2005
Prevalence of Fabry disease in patients with cryptogenic stroke: a prospective study
Arndt Rolfs, Tobias Böttcher, Marlies Zschiesche, et al.
American Journal of Medical Genetics. Part A
|
March 10, 2009
Prosaposin deficiency and saposin B deficiency (activator-deficient metachromatic leukodystrophy): report on two patients detected by analysis of urinary sphingolipids and carrying novel PSAP gene mutations
Ladislav Kuchar, Jana Ledvinová, Martin Hrebícek, et al.
Journal of Inherited Metabolic Disease
|
August 5, 2020
Natural history of multiple sulfatase deficiency: Retrospective phenotyping and functional variant analysis to characterize an ultra-rare disease
Laura A Adang, Lars Schlotawa, Samuel Groeschel, et al.
Page
of 3
Search research articles
Search
Showing results (21-30 of 30) with videos related to
Sort By:
Page
of 3
You have reached the last page of results.
This site can display upto 30 results.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society
|
January 23, 2017
Late onset Krabbe disease due to the new GALC p.Ala543Pro mutation, with intriguingly high residual GALC activity in vitro
Inge Krägeloh-Mann, Klaus Harzer, Kevin Rostásy, et al.
Human Mutation
|
December 26, 2001
NPC1: Complete genomic sequence, mutation analysis, and characterization of haplotypes
Peter Bauer, Rupert Knoblich, Claudia Bauer, et al.
JIMD Reports
|
March 17, 2021
Long-term disease course of two patients with multiple sulfatase deficiency differs from metachromatic leukodystrophy in a broad cohort
Stefanie Beck-Wödl, Christiane Kehrer, Klaus Harzer, et al.
JIMD Reports
|
July 13, 2022
Extremely low arylsulfatase A enzyme activity does not necessarily cause symptoms: A long-term follow-up and review of the literature
Lucia Laugwitz, Vidiyaah Santhanakumaran, Mareike Spieker, et al.
Molecular Genetics and Metabolism
|
February 5, 2023
Corrigendum to "Predicting clinical phenotypes of metachromatic leukodystrophy based on the arylsulfatase A activity and the ARSA genotype? - Chances and challenges" Mol Genet Metab/Vol 137/Issue 3/2022/ 273-282
Vidiyaah Santhanakumaran, Samuel Groeschel, Klaus Harzer, et al.
Molecular Genetics and Metabolism
|
October 14, 2022
Predicting clinical phenotypes of metachromatic leukodystrophy based on the arylsulfatase A activity and the ARSA genotype? - Chances and challenges
Vidiyaah Santhanakumaran, Samuel Groeschel, Klaus Harzer, et al.
Human Mutation
|
August 2, 2017
Enzymatic characterization of novel arylsulfatase A variants using human arylsulfatase A-deficient immortalized mesenchymal stromal cells
Judith Böhringer, René Santer, Neele Schumacher, et al.
Lancet (London, England)
|
November 22, 2005
Prevalence of Fabry disease in patients with cryptogenic stroke: a prospective study
Arndt Rolfs, Tobias Böttcher, Marlies Zschiesche, et al.
American Journal of Medical Genetics. Part A
|
March 10, 2009
Prosaposin deficiency and saposin B deficiency (activator-deficient metachromatic leukodystrophy): report on two patients detected by analysis of urinary sphingolipids and carrying novel PSAP gene mutations
Ladislav Kuchar, Jana Ledvinová, Martin Hrebícek, et al.
Journal of Inherited Metabolic Disease
|
August 5, 2020
Natural history of multiple sulfatase deficiency: Retrospective phenotyping and functional variant analysis to characterize an ultra-rare disease
Laura A Adang, Lars Schlotawa, Samuel Groeschel, et al.
Page
of 3