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Neurology. Genetics
|
February 12, 2020
Clinical spectrum of POLR3-related leukodystrophy caused by biallelic <i>POLR1C</i> pathogenic variants
Laurence Gauquelin, Ferdy K Cayami, László Sztriha, et al.
Molecular Genetics and Metabolism
|
September 3, 2017
Revised consensus statement on the preventive and symptomatic care of patients with leukodystrophies
Laura A Adang, Omar Sherbini, Laura Ball, et al.
American Journal of Human Genetics
|
April 16, 2019
Bi-allelic CSF1R Mutations Cause Skeletal Dysplasia of Dysosteosclerosis-Pyle Disease Spectrum and Degenerative Encephalopathy with Brain Malformation
Long Guo, Débora Romeo Bertola, Asako Takanohashi, et al.
Medrxiv : the Preprint Server for Health Sciences
|
August 12, 2025
<i>BLOC1S1</i> variants cause lysosomal and autophagic defects resulting in a hypomyelinating leukodystrophy with epileptic encephalopathy
Raffaella De Pace, Carlos Dominguez Gonzalez, Chad D Williamson, et al.
American Journal of Human Genetics
|
March 26, 2026
BLOC1S1 variants cause lysosomal and autophagic defects resulting in a hypomyelinating leukodystrophy with epileptic encephalopathy
Raffaella De Pace, Carlos A Dominguez Gonzalez, Chad D Williamson, et al.
Neurology
|
November 13, 2025
Consensus-Based Expert Recommendations for Diagnosis and Clinical Management of Vanishing White Matter
Romy J van Voorst, Daphne H Schoenmakers, Joshua L Bonkowsky, 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.
Medrxiv : the Preprint Server for Health Sciences
|
April 10, 2026
<i>PTPN1</i> -related autoinflammation is a common cause of Aicardi-Goutières Syndrome with reduced penetrance
Daniel G Calame, Emma K Wiener, Francesco Gavazzi, et al.
Human Mutation
|
April 26, 2019
Mutation update for the SATB2 gene
Yuri A Zarate, Katherine A Bosanko, Aisling R Caffrey, et al.
HGG Advances
|
April 8, 2025
Multiple molecular diagnoses identified through genome sequencing in individuals with suspected rare disease
Alka Malhotra, Erin Thorpe, Alison J Coffey, et al.
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of 22
Search research articles
Search
Showing results (191-200 of 217) with videos related to
Sort By:
Page
of 22
Neurology. Genetics
|
February 12, 2020
Clinical spectrum of POLR3-related leukodystrophy caused by biallelic <i>POLR1C</i> pathogenic variants
Laurence Gauquelin, Ferdy K Cayami, László Sztriha, et al.
Molecular Genetics and Metabolism
|
September 3, 2017
Revised consensus statement on the preventive and symptomatic care of patients with leukodystrophies
Laura A Adang, Omar Sherbini, Laura Ball, et al.
American Journal of Human Genetics
|
April 16, 2019
Bi-allelic CSF1R Mutations Cause Skeletal Dysplasia of Dysosteosclerosis-Pyle Disease Spectrum and Degenerative Encephalopathy with Brain Malformation
Long Guo, Débora Romeo Bertola, Asako Takanohashi, et al.
Medrxiv : the Preprint Server for Health Sciences
|
August 12, 2025
<i>BLOC1S1</i> variants cause lysosomal and autophagic defects resulting in a hypomyelinating leukodystrophy with epileptic encephalopathy
Raffaella De Pace, Carlos Dominguez Gonzalez, Chad D Williamson, et al.
American Journal of Human Genetics
|
March 26, 2026
BLOC1S1 variants cause lysosomal and autophagic defects resulting in a hypomyelinating leukodystrophy with epileptic encephalopathy
Raffaella De Pace, Carlos A Dominguez Gonzalez, Chad D Williamson, et al.
Neurology
|
November 13, 2025
Consensus-Based Expert Recommendations for Diagnosis and Clinical Management of Vanishing White Matter
Romy J van Voorst, Daphne H Schoenmakers, Joshua L Bonkowsky, 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.
Medrxiv : the Preprint Server for Health Sciences
|
April 10, 2026
<i>PTPN1</i> -related autoinflammation is a common cause of Aicardi-Goutières Syndrome with reduced penetrance
Daniel G Calame, Emma K Wiener, Francesco Gavazzi, et al.
Human Mutation
|
April 26, 2019
Mutation update for the SATB2 gene
Yuri A Zarate, Katherine A Bosanko, Aisling R Caffrey, et al.
HGG Advances
|
April 8, 2025
Multiple molecular diagnoses identified through genome sequencing in individuals with suspected rare disease
Alka Malhotra, Erin Thorpe, Alison J Coffey, et al.
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of 22