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NPJ Genomic Medicine
|
May 25, 2026
Expanding the phenotypic spectrum of FGF12-epilepsy-does prompt precision therapy affect outcomes?
Leo Arkush, Kristina Karandasheva, Frédérique Ouellet, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 29, 2020
Cell-based analysis of CAD variants identifies individuals likely to benefit from uridine therapy
Francisco Del Caño-Ochoa, Bobby G Ng, Malak Abedalthagafi, et al.
Cell
|
June 13, 2024
A pseudoautosomal glycosylation disorder prompts the revision of dolichol biosynthesis
Matthew P Wilson, Takfarinas Kentache, Charlotte R Althoff, et al.
Cell
|
May 31, 2024
A pseudoautosomal glycosylation disorder prompts the revision of dolichol biosynthesis
Matthew P Wilson, Takfarinas Kentache, Charlotte R Althoff, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
October 15, 2025
Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study
David Cheerie, Marlen C Lauffer, Logan Newton, et al.
BMJ Open
|
August 10, 2025
TRIAGE-GS: protocol for a randomised controlled trial of a genomics-first approach to rare disease diagnosis for patients awaiting assessment by a clinical geneticist
Kaitlin J Stanley, Caitlin Chisholm, Meredith K Gillespie, et al.
Frontiers in Molecular Neuroscience
|
March 3, 2020
Expanding Clinical Presentations Due to Variations in THOC2 mRNA Nuclear Export Factor
Raman Kumar, Elizabeth Palmer, Alison E Gardner, et al.
JAMA Network Open
|
September 22, 2020
Genome Sequencing as a Diagnostic Test in Children With Unexplained Medical Complexity
Gregory Costain, Susan Walker, Maria Marano, et al.
NPJ Genomic Medicine
|
February 27, 2025
International Precision Child Health Partnership (IPCHiP): an initiative to accelerate discovery and improve outcomes in rare pediatric disease
Katherine B Howell, Susan M White, Amy McTague, et al.
JAMA Neurology
|
May 4, 2026
Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies
Emma Sherrill, David Cheerie, Cara J Beck, et al.
Page
of 15
Search research articles
Search
Showing results (101-110 of 144) with videos related to
Sort By:
Page
of 15
NPJ Genomic Medicine
|
May 25, 2026
Expanding the phenotypic spectrum of FGF12-epilepsy-does prompt precision therapy affect outcomes?
Leo Arkush, Kristina Karandasheva, Frédérique Ouellet, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 29, 2020
Cell-based analysis of CAD variants identifies individuals likely to benefit from uridine therapy
Francisco Del Caño-Ochoa, Bobby G Ng, Malak Abedalthagafi, et al.
Cell
|
June 13, 2024
A pseudoautosomal glycosylation disorder prompts the revision of dolichol biosynthesis
Matthew P Wilson, Takfarinas Kentache, Charlotte R Althoff, et al.
Cell
|
May 31, 2024
A pseudoautosomal glycosylation disorder prompts the revision of dolichol biosynthesis
Matthew P Wilson, Takfarinas Kentache, Charlotte R Althoff, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
October 15, 2025
Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study
David Cheerie, Marlen C Lauffer, Logan Newton, et al.
BMJ Open
|
August 10, 2025
TRIAGE-GS: protocol for a randomised controlled trial of a genomics-first approach to rare disease diagnosis for patients awaiting assessment by a clinical geneticist
Kaitlin J Stanley, Caitlin Chisholm, Meredith K Gillespie, et al.
Frontiers in Molecular Neuroscience
|
March 3, 2020
Expanding Clinical Presentations Due to Variations in THOC2 mRNA Nuclear Export Factor
Raman Kumar, Elizabeth Palmer, Alison E Gardner, et al.
JAMA Network Open
|
September 22, 2020
Genome Sequencing as a Diagnostic Test in Children With Unexplained Medical Complexity
Gregory Costain, Susan Walker, Maria Marano, et al.
NPJ Genomic Medicine
|
February 27, 2025
International Precision Child Health Partnership (IPCHiP): an initiative to accelerate discovery and improve outcomes in rare pediatric disease
Katherine B Howell, Susan M White, Amy McTague, et al.
JAMA Neurology
|
May 4, 2026
Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies
Emma Sherrill, David Cheerie, Cara J Beck, et al.
Page
of 15