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JIMD Reports
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February 28, 2016
Clinical Evolution After Enzyme Replacement Therapy in Twins with the Severe Form of Maroteaux-Lamy Syndrome
M Pineda, M O'Callaghan, A Fernandez Lopez, et al.
JIMD Reports
|
November 4, 2015
Normoglycemic Ketonemia as Biochemical Presentation in Ketotic Glycogen Storage Disease
Irene J Hoogeveen, Rixt M van der Ende, Francjan J van Spronsen, et al.
JIMD Reports
|
November 6, 2015
Cerebral Lipid Accumulation Detected by MRS in a Child with Carnitine Palmitoyltransferase 2 Deficiency: A Case Report and Review of the Literature on Genetic Etiologies of Lipid Peaks on MRS
Carlos R Ferreira, Molly H Silber, Taeun Chang, et al.
JIMD Reports
|
November 6, 2015
LARS2 Variants Associated with Hydrops, Lactic Acidosis, Sideroblastic Anemia, and Multisystem Failure
Lisa G Riley, Joëlle Rudinger-Thirion, Klaus Schmitz-Abe, et al.
JIMD Reports
|
November 6, 2015
Lower Urinary Tract Symptoms and Incontinence in Children with Pompe Disease
Divya Ajay, Erin R McNamara, Stephanie Austin, et al.
JIMD Reports
|
October 30, 2015
The Nutritional Intake of Patients with Organic Acidaemias on Enteral Tube Feeding: Can We Do Better?
Anne Daly, S Evans, A Gerrard, et al.
JIMD Reports
|
April 20, 2018
Effectiveness of Early Hematopoietic Stem Cell Transplantation in Preventing Neurocognitive Decline in Mucopolysaccharidosis Type II: A Case Series
A Selvanathan, C Ellaway, C Wilson, et al.
JIMD Reports
|
July 13, 2022
Two successful pregnancies and first use of empagliflozin during pregnancy in glycogen storage disease type Ib
Sarah Catharina Grünert, Stefanie Rosenbaum-Fabian, Anke Schumann, et al.
JIMD Reports
|
July 13, 2022
Glutaric aciduria type 1: Diagnosis, clinical features and long-term outcome in a large cohort of 34 Irish patients
Lydia Healy, Meabh O'Shea, Jennifer McNulty, et al.
JIMD Reports
|
July 13, 2022
Association of alkaptonuria and low dose nitisinone therapy with cataract formation in a large cohort of patients
Mohammad S Z Ahmad, Mahmoud Ahmed, Milad Khedr, et al.
Page
of 125
Search research articles
Search
Showing results (951-960 of 1,250) with videos related to
Sort By:
Page
of 125
JIMD Reports
|
February 28, 2016
Clinical Evolution After Enzyme Replacement Therapy in Twins with the Severe Form of Maroteaux-Lamy Syndrome
M Pineda, M O'Callaghan, A Fernandez Lopez, et al.
JIMD Reports
|
November 4, 2015
Normoglycemic Ketonemia as Biochemical Presentation in Ketotic Glycogen Storage Disease
Irene J Hoogeveen, Rixt M van der Ende, Francjan J van Spronsen, et al.
JIMD Reports
|
November 6, 2015
Cerebral Lipid Accumulation Detected by MRS in a Child with Carnitine Palmitoyltransferase 2 Deficiency: A Case Report and Review of the Literature on Genetic Etiologies of Lipid Peaks on MRS
Carlos R Ferreira, Molly H Silber, Taeun Chang, et al.
JIMD Reports
|
November 6, 2015
LARS2 Variants Associated with Hydrops, Lactic Acidosis, Sideroblastic Anemia, and Multisystem Failure
Lisa G Riley, Joëlle Rudinger-Thirion, Klaus Schmitz-Abe, et al.
JIMD Reports
|
November 6, 2015
Lower Urinary Tract Symptoms and Incontinence in Children with Pompe Disease
Divya Ajay, Erin R McNamara, Stephanie Austin, et al.
JIMD Reports
|
October 30, 2015
The Nutritional Intake of Patients with Organic Acidaemias on Enteral Tube Feeding: Can We Do Better?
Anne Daly, S Evans, A Gerrard, et al.
JIMD Reports
|
April 20, 2018
Effectiveness of Early Hematopoietic Stem Cell Transplantation in Preventing Neurocognitive Decline in Mucopolysaccharidosis Type II: A Case Series
A Selvanathan, C Ellaway, C Wilson, et al.
JIMD Reports
|
July 13, 2022
Two successful pregnancies and first use of empagliflozin during pregnancy in glycogen storage disease type Ib
Sarah Catharina Grünert, Stefanie Rosenbaum-Fabian, Anke Schumann, et al.
JIMD Reports
|
July 13, 2022
Glutaric aciduria type 1: Diagnosis, clinical features and long-term outcome in a large cohort of 34 Irish patients
Lydia Healy, Meabh O'Shea, Jennifer McNulty, et al.
JIMD Reports
|
July 13, 2022
Association of alkaptonuria and low dose nitisinone therapy with cataract formation in a large cohort of patients
Mohammad S Z Ahmad, Mahmoud Ahmed, Milad Khedr, et al.
Page
of 125