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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.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society
|
June 7, 2025
Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy
Daphne H Schoenmakers, Marije A B C Asbreuk, Tamara Martin, et al.
Molecular Genetics and Metabolism
|
July 4, 2024
Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophy
Laura Ann Adang, Samuel Groeschel, Chloe Grzyb, et al.
Nature Communications
|
February 14, 2026
Loss-of-function variants in the CAPN1 activator CD99L2 cause X-linked spastic ataxia
Benita Menden, Rana D Incebacak Eltemur, German Demidov, et al.
Nature Genetics
|
July 22, 2024
Next-generation phenotyping integrated in a national framework for patients with ultrarare disorders improves genetic diagnostics and yields new molecular findings
Axel Schmidt, Magdalena Danyel, Kathrin Grundmann, et al.
Nature Genetics
|
June 24, 2025
Author Correction: Next-generation phenotyping integrated in a national framework for patients with ultrarare disorders improves genetic diagnostics and yields new molecular findings
Axel Schmidt, Magdalena Danyel, Kathrin Grundmann, et al.
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Search research articles
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Showing results (21-30 of 26) with videos related to
Sort By:
Page
of 3
You have reached the last page of results.
This site can display upto 26 results.
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.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society
|
June 7, 2025
Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy
Daphne H Schoenmakers, Marije A B C Asbreuk, Tamara Martin, et al.
Molecular Genetics and Metabolism
|
July 4, 2024
Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophy
Laura Ann Adang, Samuel Groeschel, Chloe Grzyb, et al.
Nature Communications
|
February 14, 2026
Loss-of-function variants in the CAPN1 activator CD99L2 cause X-linked spastic ataxia
Benita Menden, Rana D Incebacak Eltemur, German Demidov, et al.
Nature Genetics
|
July 22, 2024
Next-generation phenotyping integrated in a national framework for patients with ultrarare disorders improves genetic diagnostics and yields new molecular findings
Axel Schmidt, Magdalena Danyel, Kathrin Grundmann, et al.
Nature Genetics
|
June 24, 2025
Author Correction: Next-generation phenotyping integrated in a national framework for patients with ultrarare disorders improves genetic diagnostics and yields new molecular findings
Axel Schmidt, Magdalena Danyel, Kathrin Grundmann, et al.
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of 3