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Pediatric Research
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June 23, 2009
Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease
Priya S Kishnani, Deya Corzo, Nancy D Leslie, et al.
Journal of Medical Genetics
|
July 17, 2023
Exome sequencing links the SUMO protease SENP7 with fatal arthrogryposis multiplex congenita, early respiratory failure and neutropenia
Nadra Samra, Nicolette S Jansen, Ilham Morani, et al.
Journal of Medical Genetics
|
August 6, 2025
Exploring the unique characteristics of genes with dual autosomal dominant and recessive inheritance: mechanisms, phenotypes and candidate identification
Shlomit Ezer, Tal Sido, Jonathan Rips, et al.
Human Mutation
|
January 10, 2018
Clinical, biochemical, and genetic features associated with VARS2-related mitochondrial disease
Francesco Bruni, Ivano Di Meo, Emanuele Bellacchio, et al.
Neurology. Genetics
|
March 27, 2018
<i>ACO2</i> homozygous missense mutation associated with complicated hereditary spastic paraplegia
Christian G Bouwkamp, Zaid Afawi, Aviva Fattal-Valevski, et al.
Human Mutation
|
October 15, 2008
Molecular investigations to improve diagnostic accuracy in patients with ARC syndrome
Andrew R Cullinane, Anna Straatman-Iwanowska, Jeong K Seo, et al.
American Journal of Ophthalmology
|
November 16, 2023
Lisch Epithelial Corneal Dystrophy Is Caused by Heterozygous Loss-of-Function Variants in MCOLN1
Karynne Patterson, Jessica X Chong, Doug D Chung, et al.
Human Genetics
|
August 10, 2006
Clinical and molecular genetic features of ARC syndrome
Paul Gissen, Louise Tee, Colin A Johnson, et al.
Plos Genetics
|
January 14, 2012
Autosomal recessive dilated cardiomyopathy due to DOLK mutations results from abnormal dystroglycan O-mannosylation
Dirk J Lefeber, Arjan P M de Brouwer, Eva Morava, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
March 17, 2009
Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease
Marc Nicolino, Barry Byrne, J Edmund Wraith, et al.
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of 13
Search research articles
Search
Showing results (91-100 of 127) with videos related to
Sort By:
Page
of 13
Pediatric Research
|
June 23, 2009
Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease
Priya S Kishnani, Deya Corzo, Nancy D Leslie, et al.
Journal of Medical Genetics
|
July 17, 2023
Exome sequencing links the SUMO protease SENP7 with fatal arthrogryposis multiplex congenita, early respiratory failure and neutropenia
Nadra Samra, Nicolette S Jansen, Ilham Morani, et al.
Journal of Medical Genetics
|
August 6, 2025
Exploring the unique characteristics of genes with dual autosomal dominant and recessive inheritance: mechanisms, phenotypes and candidate identification
Shlomit Ezer, Tal Sido, Jonathan Rips, et al.
Human Mutation
|
January 10, 2018
Clinical, biochemical, and genetic features associated with VARS2-related mitochondrial disease
Francesco Bruni, Ivano Di Meo, Emanuele Bellacchio, et al.
Neurology. Genetics
|
March 27, 2018
<i>ACO2</i> homozygous missense mutation associated with complicated hereditary spastic paraplegia
Christian G Bouwkamp, Zaid Afawi, Aviva Fattal-Valevski, et al.
Human Mutation
|
October 15, 2008
Molecular investigations to improve diagnostic accuracy in patients with ARC syndrome
Andrew R Cullinane, Anna Straatman-Iwanowska, Jeong K Seo, et al.
American Journal of Ophthalmology
|
November 16, 2023
Lisch Epithelial Corneal Dystrophy Is Caused by Heterozygous Loss-of-Function Variants in MCOLN1
Karynne Patterson, Jessica X Chong, Doug D Chung, et al.
Human Genetics
|
August 10, 2006
Clinical and molecular genetic features of ARC syndrome
Paul Gissen, Louise Tee, Colin A Johnson, et al.
Plos Genetics
|
January 14, 2012
Autosomal recessive dilated cardiomyopathy due to DOLK mutations results from abnormal dystroglycan O-mannosylation
Dirk J Lefeber, Arjan P M de Brouwer, Eva Morava, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
March 17, 2009
Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease
Marc Nicolino, Barry Byrne, J Edmund Wraith, et al.
Page
of 13