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International Journal of Environmental Research and Public Health
|
May 25, 2024
Oral Health-Related Quality of Life in Rare Disorders of Congenital Facial Weakness
Denise K Liberton, Konstantinia Almpani, Rashmi Mishra, et al.
The Cleft Palate-Craniofacial Journal : Official Publication of the American Cleft Palate-Craniofacial Association
|
July 4, 2025
Multimodality Craniofacial Phenotyping of Congenital Facial Weakness Disorders
Konstantinia Almpani, Katelin R Devine, Denise K Liberton, et al.
Journal of Inherited Metabolic Disease
|
May 1, 2022
Diagnosis and discovery: Insights from the NIH Undiagnosed Diseases Program
Carolina Montano, Thomas Cassini, Shira G Ziegler, et al.
Molecular Metabolism
|
October 1, 2016
Defects in muscle branched-chain amino acid oxidation contribute to impaired lipid metabolism
Carles Lerin, Allison B Goldfine, Tanner Boes, et al.
Human Gene Therapy
|
February 20, 2025
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program
Richa Madan Lomash, Jean Dehdashti, Oleg A Shchelochkov, et al.
Molecular Therapy. Methods & Clinical Development
|
September 6, 2021
ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia
Petr O Ilyinskii, Alicia M Michaud, Gina L Rizzo, et al.
Human Gene Therapy
|
June 19, 2026
Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory Meetings
Rodica Stan, Richa Madan Lomash, Oleg A Shchelochkov, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 19, 2021
Severity modeling of propionic acidemia using clinical and laboratory biomarkers
Oleg A Shchelochkov, Irini Manoli, Paul Juneau, et al.
American Journal of Human Genetics
|
September 10, 2013
An X-linked cobalamin disorder caused by mutations in transcriptional coregulator HCFC1
Hung-Chun Yu, Jennifer L Sloan, Gunter Scharer, et al.
American Journal of Medical Genetics. Part A
|
August 5, 2017
Identification of STAC3 variants in non-Native American families with overlapping features of Carey-Fineman-Ziter syndrome and Moebius syndrome
Aida Telegrafi, Bryn D Webb, Sarah M Robbins, et al.
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of 8
Search research articles
Search
Showing results (51-60 of 72) with videos related to
Sort By:
Page
of 8
International Journal of Environmental Research and Public Health
|
May 25, 2024
Oral Health-Related Quality of Life in Rare Disorders of Congenital Facial Weakness
Denise K Liberton, Konstantinia Almpani, Rashmi Mishra, et al.
The Cleft Palate-Craniofacial Journal : Official Publication of the American Cleft Palate-Craniofacial Association
|
July 4, 2025
Multimodality Craniofacial Phenotyping of Congenital Facial Weakness Disorders
Konstantinia Almpani, Katelin R Devine, Denise K Liberton, et al.
Journal of Inherited Metabolic Disease
|
May 1, 2022
Diagnosis and discovery: Insights from the NIH Undiagnosed Diseases Program
Carolina Montano, Thomas Cassini, Shira G Ziegler, et al.
Molecular Metabolism
|
October 1, 2016
Defects in muscle branched-chain amino acid oxidation contribute to impaired lipid metabolism
Carles Lerin, Allison B Goldfine, Tanner Boes, et al.
Human Gene Therapy
|
February 20, 2025
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program
Richa Madan Lomash, Jean Dehdashti, Oleg A Shchelochkov, et al.
Molecular Therapy. Methods & Clinical Development
|
September 6, 2021
ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia
Petr O Ilyinskii, Alicia M Michaud, Gina L Rizzo, et al.
Human Gene Therapy
|
June 19, 2026
Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory Meetings
Rodica Stan, Richa Madan Lomash, Oleg A Shchelochkov, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 19, 2021
Severity modeling of propionic acidemia using clinical and laboratory biomarkers
Oleg A Shchelochkov, Irini Manoli, Paul Juneau, et al.
American Journal of Human Genetics
|
September 10, 2013
An X-linked cobalamin disorder caused by mutations in transcriptional coregulator HCFC1
Hung-Chun Yu, Jennifer L Sloan, Gunter Scharer, et al.
American Journal of Medical Genetics. Part A
|
August 5, 2017
Identification of STAC3 variants in non-Native American families with overlapping features of Carey-Fineman-Ziter syndrome and Moebius syndrome
Aida Telegrafi, Bryn D Webb, Sarah M Robbins, et al.
Page
of 8