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Neurology. Genetics
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May 31, 2021
Diagnosing Mitochondrial Disorders Remains Challenging in the Omics Era
Patrick Forny, Emma Footitt, James E Davison, et al.
Analytical Chemistry
|
November 6, 2015
Proteomic Discovery and Development of a Multiplexed Targeted MRM-LC-MS/MS Assay for Urine Biomarkers of Extracellular Matrix Disruption in Mucopolysaccharidoses I, II, and VI
Wendy E Heywood, Stephane Camuzeaux, Ivan Doykov, et al.
Brain : a Journal of Neurology
|
September 9, 2016
Advantages and pitfalls of an extended gene panel for investigating complex neurometabolic phenotypes
Emma S Reid, Apostolos Papandreou, Suzanne Drury, et al.
Molecular Genetics and Metabolism
|
July 15, 2025
Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial
Eugen Mengel, Rosalia M Da Riol, Mireia Del Toro, et al.
Journal of Medical Virology
|
August 20, 2021
Evolution of viral variants in remdesivir-treated and untreated SARS-CoV-2-infected pediatrics patients
Florencia A T Boshier, Juanita Pang, Justin Penner, et al.
Journal of Inherited Metabolic Disease
|
February 17, 2021
Guidelines for the diagnosis and management of methylmalonic acidaemia and propionic acidaemia: First revision
Patrick Forny, Friederike Hörster, Diana Ballhausen, et al.
Orphanet Journal of Rare Diseases
|
November 24, 2020
Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study
Eugen Mengel, Bruno Bembi, Mireia Del Toro, et al.
JAMA Ophthalmology
|
March 3, 2017
Association of Steroid 5α-Reductase Type 3 Congenital Disorder of Glycosylation With Early-Onset Retinal Dystrophy
Rachel L Taylor, Gavin Arno, James A Poulter, et al.
Molecular Genetics and Metabolism
|
December 21, 2020
Phosphoglucomutase-1 deficiency: Early presentation, metabolic management and detection in neonatal blood spots
Federica Conte, Eva Morava, Nurulamin Abu Bakar, et al.
Journal of Inherited Metabolic Disease
|
December 5, 2024
Clinical severity and cardiac phenotype in phosphomannomutase 2-congenital disorders of glycosylation : Insights into genetics and management recommendations
Veronika Holubova, Rita Barone, Stephanie Grunewald, et al.
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Search research articles
Search
Showing results (31-40 of 59) with videos related to
Sort By:
Page
of 6
Neurology. Genetics
|
May 31, 2021
Diagnosing Mitochondrial Disorders Remains Challenging in the Omics Era
Patrick Forny, Emma Footitt, James E Davison, et al.
Analytical Chemistry
|
November 6, 2015
Proteomic Discovery and Development of a Multiplexed Targeted MRM-LC-MS/MS Assay for Urine Biomarkers of Extracellular Matrix Disruption in Mucopolysaccharidoses I, II, and VI
Wendy E Heywood, Stephane Camuzeaux, Ivan Doykov, et al.
Brain : a Journal of Neurology
|
September 9, 2016
Advantages and pitfalls of an extended gene panel for investigating complex neurometabolic phenotypes
Emma S Reid, Apostolos Papandreou, Suzanne Drury, et al.
Molecular Genetics and Metabolism
|
July 15, 2025
Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial
Eugen Mengel, Rosalia M Da Riol, Mireia Del Toro, et al.
Journal of Medical Virology
|
August 20, 2021
Evolution of viral variants in remdesivir-treated and untreated SARS-CoV-2-infected pediatrics patients
Florencia A T Boshier, Juanita Pang, Justin Penner, et al.
Journal of Inherited Metabolic Disease
|
February 17, 2021
Guidelines for the diagnosis and management of methylmalonic acidaemia and propionic acidaemia: First revision
Patrick Forny, Friederike Hörster, Diana Ballhausen, et al.
Orphanet Journal of Rare Diseases
|
November 24, 2020
Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study
Eugen Mengel, Bruno Bembi, Mireia Del Toro, et al.
JAMA Ophthalmology
|
March 3, 2017
Association of Steroid 5α-Reductase Type 3 Congenital Disorder of Glycosylation With Early-Onset Retinal Dystrophy
Rachel L Taylor, Gavin Arno, James A Poulter, et al.
Molecular Genetics and Metabolism
|
December 21, 2020
Phosphoglucomutase-1 deficiency: Early presentation, metabolic management and detection in neonatal blood spots
Federica Conte, Eva Morava, Nurulamin Abu Bakar, et al.
Journal of Inherited Metabolic Disease
|
December 5, 2024
Clinical severity and cardiac phenotype in phosphomannomutase 2-congenital disorders of glycosylation : Insights into genetics and management recommendations
Veronika Holubova, Rita Barone, Stephanie Grunewald, et al.
Page
of 6