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JIMD Reports
|
November 12, 2013
Secondary Mitochondrial Respiratory Chain Defect Can Delay Accurate PFIC2 Diagnosis
Anne Davit-Spraul, Marine Beinat, Dominique Debray, et al.
JIMD Reports
|
November 14, 2013
Dietary habits and metabolic control in adolescents and young adults with phenylketonuria: self-imposed protein restriction may be harmful
A M Das, K Goedecke, U Meyer, et al.
JIMD Reports
|
April 13, 2026
Long-Term Safety and Efficacy of Pegvaliase in Japanese Adults With Phenylketonuria: Final Results of a Phase III Trial
Yoko Nakajima, Mika Ishige, Tetsuya Ito, et al.
JIMD Reports
|
September 10, 2019
The clinical profiles of female patients with Fabry disease in Latin America: A Fabry Registry analysis of natural history data from 169 patients based on enzyme replacement therapy status
Ana M Martins, Gustavo Cabrera, Fernando Molt, et al.
JIMD Reports
|
February 23, 2013
Successful weight loss in two adult patients diagnosed with late-onset long-chain Fatty Acid oxidation defect
H Zweers, C Timmer, E Rasmussen, et al.
JIMD Reports
|
April 8, 2026
Unifying the Communities of Early-Onset Glycogen Storage Disease Type IV and Adult Polyglucosan Body Disease Through a Genetic Prevalence Study of <i>GBE1</i>-Related Disease
Rebecca L Koch, H Orhan Akman, Erin Chown, et al.
JIMD Reports
|
March 2, 2026
Abnormal Newborn Screening Resembling Carnitine Palmitoyltransferase 1a Deficiency in Three Patients With <i>COASY</i> Protein Associated Neurodegeneration
Matthew Lynch, Sophie Manoy, Claire Murray, et al.
JIMD Reports
|
February 25, 2026
Clinical Profiles, Genetic Variants, and Neurodevelopmental Outcomes Following Liver Transplantation in Maple Syrup Urine Disease: A Study From Palestine
Reham Khalaf-Nazzal, Huthaifa Haj-Ahmad, Jana Zaid, et al.
JIMD Reports
|
February 25, 2026
Intrathecal idursulfase-IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single-arm, open-label, phase 2/3 substudy and extension
Joseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
JIMD Reports
|
March 9, 2026
Alkaline Phosphatase and Infantile GM1 Gangliosidosis: A Simple Biomarker for a Complex Disease?
Laura Fiori, Massimiliano Turzi, Veronica Maria Tagi, et al.
Page
of 127
Search research articles
Search
Showing results (221-230 of 1,270) with videos related to
Sort By:
Page
of 127
JIMD Reports
|
November 12, 2013
Secondary Mitochondrial Respiratory Chain Defect Can Delay Accurate PFIC2 Diagnosis
Anne Davit-Spraul, Marine Beinat, Dominique Debray, et al.
JIMD Reports
|
November 14, 2013
Dietary habits and metabolic control in adolescents and young adults with phenylketonuria: self-imposed protein restriction may be harmful
A M Das, K Goedecke, U Meyer, et al.
JIMD Reports
|
April 13, 2026
Long-Term Safety and Efficacy of Pegvaliase in Japanese Adults With Phenylketonuria: Final Results of a Phase III Trial
Yoko Nakajima, Mika Ishige, Tetsuya Ito, et al.
JIMD Reports
|
September 10, 2019
The clinical profiles of female patients with Fabry disease in Latin America: A Fabry Registry analysis of natural history data from 169 patients based on enzyme replacement therapy status
Ana M Martins, Gustavo Cabrera, Fernando Molt, et al.
JIMD Reports
|
February 23, 2013
Successful weight loss in two adult patients diagnosed with late-onset long-chain Fatty Acid oxidation defect
H Zweers, C Timmer, E Rasmussen, et al.
JIMD Reports
|
April 8, 2026
Unifying the Communities of Early-Onset Glycogen Storage Disease Type IV and Adult Polyglucosan Body Disease Through a Genetic Prevalence Study of <i>GBE1</i>-Related Disease
Rebecca L Koch, H Orhan Akman, Erin Chown, et al.
JIMD Reports
|
March 2, 2026
Abnormal Newborn Screening Resembling Carnitine Palmitoyltransferase 1a Deficiency in Three Patients With <i>COASY</i> Protein Associated Neurodegeneration
Matthew Lynch, Sophie Manoy, Claire Murray, et al.
JIMD Reports
|
February 25, 2026
Clinical Profiles, Genetic Variants, and Neurodevelopmental Outcomes Following Liver Transplantation in Maple Syrup Urine Disease: A Study From Palestine
Reham Khalaf-Nazzal, Huthaifa Haj-Ahmad, Jana Zaid, et al.
JIMD Reports
|
February 25, 2026
Intrathecal idursulfase-IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single-arm, open-label, phase 2/3 substudy and extension
Joseph Muenzer, Barbara K Burton, Paul Harmatz, et al.
JIMD Reports
|
March 9, 2026
Alkaline Phosphatase and Infantile GM1 Gangliosidosis: A Simple Biomarker for a Complex Disease?
Laura Fiori, Massimiliano Turzi, Veronica Maria Tagi, et al.
Page
of 127