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Brain : a Journal of Neurology
|
July 24, 2019
Cohesin complex-associated holoprosencephaly
Paul Kruszka, Seth I Berger, Valentina Casa, et al.
NEJM Evidence
|
February 6, 2024
Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI
Nicola Brunetti-Pierri, Rita Ferla, Virginia Maria Ginocchio, et al.
BMJ Open
|
February 23, 2022
Families' healthcare experiences for children with inherited metabolic diseases: protocol for a mixed methods cohort study
Andrea J Chow, Ryan Iverson, Monica Lamoureux, et al.
Brain Communications
|
February 18, 2025
Biallelic <i>NDUFA13</i> variants lead to a neurodevelopmental phenotype with gradual neurological impairment
Rauan Kaiyrzhanov, Kyle Thompson, Stephanie Efthymiou, et al.
American Journal of Human Genetics
|
January 17, 2025
Discovery of a DNA methylation profile in individuals with Sifrim-Hitz-Weiss syndrome
Karim Karimi, Yael Lichtenstein, Jack Reilly, et al.
BMC Pediatrics
|
January 12, 2024
Assessing the quality and value of metabolic chart data for capturing core outcomes for pediatric medium-chain acyl-CoA dehydrogenase (MCAD) deficiency
Ryan Iverson, Monica Taljaard, Michael T Geraghty, et al.
Orphanet Journal of Rare Diseases
|
April 12, 2020
Evaluation of the quality of clinical data collection for a pan-Canadian cohort of children affected by inherited metabolic diseases: lessons learned from the Canadian Inherited Metabolic Diseases Research Network
Kylie Tingley, Monica Lamoureux, Michael Pugliese, et al.
JCI Insight
|
September 16, 2016
<i>PIK3CA</i>-associated developmental disorders exhibit distinct classes of mutations with variable expression and tissue distribution
Ghayda Mirzaa, Andrew E Timms, Valerio Conti, et al.
American Journal of Human Genetics
|
January 5, 2024
A syndromic neurodevelopmental disorder caused by rare variants in PPFIA3
Maimuna S Paul, Sydney L Michener, Hongling Pan, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
October 20, 2023
Clinico-biological refinement of BCL11B-related disorder and identification of an episignature: A series of 20 unreported individuals
Quentin Sabbagh, Sadegheh Haghshenas, Juliette Piard, et al.
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of 12
Search research articles
Search
Showing results (101-110 of 114) with videos related to
Sort By:
Page
of 12
Brain : a Journal of Neurology
|
July 24, 2019
Cohesin complex-associated holoprosencephaly
Paul Kruszka, Seth I Berger, Valentina Casa, et al.
NEJM Evidence
|
February 6, 2024
Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI
Nicola Brunetti-Pierri, Rita Ferla, Virginia Maria Ginocchio, et al.
BMJ Open
|
February 23, 2022
Families' healthcare experiences for children with inherited metabolic diseases: protocol for a mixed methods cohort study
Andrea J Chow, Ryan Iverson, Monica Lamoureux, et al.
Brain Communications
|
February 18, 2025
Biallelic <i>NDUFA13</i> variants lead to a neurodevelopmental phenotype with gradual neurological impairment
Rauan Kaiyrzhanov, Kyle Thompson, Stephanie Efthymiou, et al.
American Journal of Human Genetics
|
January 17, 2025
Discovery of a DNA methylation profile in individuals with Sifrim-Hitz-Weiss syndrome
Karim Karimi, Yael Lichtenstein, Jack Reilly, et al.
BMC Pediatrics
|
January 12, 2024
Assessing the quality and value of metabolic chart data for capturing core outcomes for pediatric medium-chain acyl-CoA dehydrogenase (MCAD) deficiency
Ryan Iverson, Monica Taljaard, Michael T Geraghty, et al.
Orphanet Journal of Rare Diseases
|
April 12, 2020
Evaluation of the quality of clinical data collection for a pan-Canadian cohort of children affected by inherited metabolic diseases: lessons learned from the Canadian Inherited Metabolic Diseases Research Network
Kylie Tingley, Monica Lamoureux, Michael Pugliese, et al.
JCI Insight
|
September 16, 2016
<i>PIK3CA</i>-associated developmental disorders exhibit distinct classes of mutations with variable expression and tissue distribution
Ghayda Mirzaa, Andrew E Timms, Valerio Conti, et al.
American Journal of Human Genetics
|
January 5, 2024
A syndromic neurodevelopmental disorder caused by rare variants in PPFIA3
Maimuna S Paul, Sydney L Michener, Hongling Pan, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
October 20, 2023
Clinico-biological refinement of BCL11B-related disorder and identification of an episignature: A series of 20 unreported individuals
Quentin Sabbagh, Sadegheh Haghshenas, Juliette Piard, et al.
Page
of 12