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Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 27, 2021
Expanded phenotype of AARS1-related white matter disease
Guy Helman, Marisa I Mendes, Francesco Nicita, 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.
Molecular Genetics and Metabolism
|
February 14, 2025
The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy
Francesco Gavazzi, Brittany Charsar, Eline Hamilton, 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.
Genetics
|
October 6, 2025
Mondo: Integrating Disease Terminology Across Communities
Nicole A Vasilevsky, Sabrina Toro, Nicolas Matentzoglu, et al.
The Journal of Clinical Endocrinology and Metabolism
|
October 2, 2020
Endocrine and Growth Abnormalities in 4H Leukodystrophy Caused by Variants in POLR3A, POLR3B, and POLR1C
Félixe Pelletier, Stefanie Perrier, Ferdy K Cayami, et al.
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Search research articles
Search
Showing results (31-40 of 42) with videos related to
Sort By:
Page
of 5
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
August 27, 2021
Expanded phenotype of AARS1-related white matter disease
Guy Helman, Marisa I Mendes, Francesco Nicita, 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.
Molecular Genetics and Metabolism
|
February 14, 2025
The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy
Francesco Gavazzi, Brittany Charsar, Eline Hamilton, 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.
Genetics
|
October 6, 2025
Mondo: Integrating Disease Terminology Across Communities
Nicole A Vasilevsky, Sabrina Toro, Nicolas Matentzoglu, et al.
The Journal of Clinical Endocrinology and Metabolism
|
October 2, 2020
Endocrine and Growth Abnormalities in 4H Leukodystrophy Caused by Variants in POLR3A, POLR3B, and POLR1C
Félixe Pelletier, Stefanie Perrier, Ferdy K Cayami, et al.
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of 5