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Molecular Genetics and Metabolism
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March 6, 2017
Clinical and molecular phenotyping of a child with Hermansky-Pudlak syndrome-7, an uncommon genetic type of HPS
Melanie M Bryan, Nathanial J Tolman, Karen L Simon, et al.
Orphanet Journal of Rare Diseases
|
February 23, 2019
Hermansky-Pudlak syndrome and oculocutaneous albinism in Chinese children with pigmentation defects and easy bruising
Bradley Power, Carlos R Ferreira, Dong Chen, et al.
Pigment Cell & Melanoma Research
|
September 3, 2011
Cellular and clinical report of new Griscelli syndrome type III cases
Wendy Westbroek, Aharon Klar, Andrew R Cullinane, et al.
Annals of Neurology
|
July 12, 2002
Adenine nucleotide translocator 1 deficiency associated with Sengers syndrome
Eric Z Jordens, Luigi Palmieri, Marjan Huizing, et al.
American Journal of Medical Genetics. Part A
|
May 27, 2010
Chediak-Higashi syndrome with early developmental delay resulting from paternal heterodisomy of chromosome 1
Irini Manoli, Gretchen Golas, Wendy Westbroek, et al.
Clinical Journal of the American Society of Nephrology : CJASN
|
January 8, 2013
1,25-(OH)2D-24 Hydroxylase (CYP24A1) Deficiency as a Cause of Nephrolithiasis
Galina Nesterova, May Christine Malicdan, Kaori Yasuda, et al.
Kidney International Reports
|
November 9, 2019
Rationale and Design for a Phase 1 Study of <i>N</i>-Acetylmannosamine for Primary Glomerular Diseases
Marjan Huizing, Tal Yardeni, Federico Fuentes, et al.
Molecular Genetics and Metabolism Reports
|
November 24, 2025
Profiling glycosphingolipid changes in mouse and human cellular models of lysosomal free sialic acid storage disorder
Marya S Sabir, Kostantin Dobrenis, Allisandra K Rha, et al.
The Journal of Gene Medicine
|
May 5, 2010
Hereditary inclusion body myopathy: single patient response to GNE gene Lipoplex therapy
Gregory Nemunaitis, Phillip B Maples, Chris Jay, et al.
Molecular Genetics and Metabolism
|
June 24, 2017
Safety, pharmacokinetics and sialic acid production after oral administration of N-acetylmannosamine (ManNAc) to subjects with GNE myopathy
Xin Xu, Amy Q Wang, Lea L Latham, et al.
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of 13
Search research articles
Search
Showing results (91-100 of 130) with videos related to
Sort By:
Page
of 13
Molecular Genetics and Metabolism
|
March 6, 2017
Clinical and molecular phenotyping of a child with Hermansky-Pudlak syndrome-7, an uncommon genetic type of HPS
Melanie M Bryan, Nathanial J Tolman, Karen L Simon, et al.
Orphanet Journal of Rare Diseases
|
February 23, 2019
Hermansky-Pudlak syndrome and oculocutaneous albinism in Chinese children with pigmentation defects and easy bruising
Bradley Power, Carlos R Ferreira, Dong Chen, et al.
Pigment Cell & Melanoma Research
|
September 3, 2011
Cellular and clinical report of new Griscelli syndrome type III cases
Wendy Westbroek, Aharon Klar, Andrew R Cullinane, et al.
Annals of Neurology
|
July 12, 2002
Adenine nucleotide translocator 1 deficiency associated with Sengers syndrome
Eric Z Jordens, Luigi Palmieri, Marjan Huizing, et al.
American Journal of Medical Genetics. Part A
|
May 27, 2010
Chediak-Higashi syndrome with early developmental delay resulting from paternal heterodisomy of chromosome 1
Irini Manoli, Gretchen Golas, Wendy Westbroek, et al.
Clinical Journal of the American Society of Nephrology : CJASN
|
January 8, 2013
1,25-(OH)2D-24 Hydroxylase (CYP24A1) Deficiency as a Cause of Nephrolithiasis
Galina Nesterova, May Christine Malicdan, Kaori Yasuda, et al.
Kidney International Reports
|
November 9, 2019
Rationale and Design for a Phase 1 Study of <i>N</i>-Acetylmannosamine for Primary Glomerular Diseases
Marjan Huizing, Tal Yardeni, Federico Fuentes, et al.
Molecular Genetics and Metabolism Reports
|
November 24, 2025
Profiling glycosphingolipid changes in mouse and human cellular models of lysosomal free sialic acid storage disorder
Marya S Sabir, Kostantin Dobrenis, Allisandra K Rha, et al.
The Journal of Gene Medicine
|
May 5, 2010
Hereditary inclusion body myopathy: single patient response to GNE gene Lipoplex therapy
Gregory Nemunaitis, Phillip B Maples, Chris Jay, et al.
Molecular Genetics and Metabolism
|
June 24, 2017
Safety, pharmacokinetics and sialic acid production after oral administration of N-acetylmannosamine (ManNAc) to subjects with GNE myopathy
Xin Xu, Amy Q Wang, Lea L Latham, et al.
Page
of 13