l-Carnitine and Acetyl-l-carnitine Roles and Neuroprotection in Develo** Brain

GC Ferreira, MC McKenna - Neurochemical research, 2017‏ - Springer
Abstract l-Carnitine functions to transport long chain fatty acyl-CoAs into the mitochondria for
degradation by β-oxidation. Treatment with l-carnitine can ameliorate metabolic imbalances …

Proposed recommendations for diagnosing and managing individuals with glutaric aciduria type I: second revision

N Boy, C Mühlhausen, EM Maier, J Heringer… - Journal of inherited …, 2017‏ - Springer
Glutaric aciduria type I (GA-I; synonym, glutaric acidemia type I) is a rare inherited metabolic
disease caused by deficiency of glutaryl-CoA dehydrogenase located in the catabolic …

Recommendations for diagnosing and managing individuals with glutaric aciduria type 1: third revision

N Boy, C Mühlhausen, EM Maier… - Journal of inherited …, 2023‏ - Wiley Online Library
Glutaric aciduria type 1 is a rare inherited neurometabolic disorder of lysine metabolism
caused by pathogenic gene variations in GCDH (cytogenic location: 19p13. 13), resulting in …

The phenotypic spectrum of organic acidurias and urea cycle disorders. Part 1: the initial presentation

S Kölker, AG Cazorla… - Journal of Inherited …, 2015‏ - Wiley Online Library
Background The clinical presentation of patients with organic acidurias (OAD) and urea
cycle disorders (UCD) is variable; symptoms are often non‐specific. Aims/methods To …

Diagnosis and management of glutaric aciduria type I–revised recommendations

S Kölker, E Christensen, JV Leonard… - Journal of inherited …, 2011‏ - Springer
Glutaric aciduria type I (synonym, glutaric acidemia type I) is a rare organic aciduria.
Untreated patients characteristically develop dystonia during infancy resulting in a high …

L-carnitine supplementation as a potential antioxidant therapy for inherited neurometabolic disorders

GS Ribas, CR Vargas, M Wajner - Gene, 2014‏ - Elsevier
In recent years increasing evidence has emerged suggesting that oxidative stress is
involved in the pathophysiology of a number of inherited metabolic disorders. However the …

Natural history, outcome, and treatment efficacy in children and adults with glutaryl-CoA dehydrogenase deficiency

S Kölker, SF Garbade, CR Greenberg, JV Leonard… - Pediatric …, 2006‏ - nature.com
Glutaryl-CoA dehydrogenase (GCDH) deficiency is a rare inborn disorder of l-lysine, l-
hydroxylysine, and l-tryptophan metabolism complicated by striatal damage during acute …

Exploring genotype–phenotype correlations in glutaric aciduria type 1

IME Schuurmans, B Dimitrov, J Schröter… - Journal of Inherited …, 2023‏ - Wiley Online Library
Abstract Glutaric aciduria type 1 (GA1) is a rare neurometabolic disease caused by
pathogenic variants in the gene encoding the enzyme glutaryl‐CoA dehydrogenase …

Newborn screening: a disease‐changing intervention for glutaric aciduria type 1

N Boy, K Mengler, E Thimm, KA Schiergens… - Annals of …, 2018‏ - Wiley Online Library
Objective Untreated individuals with glutaric aciduria type 1 (GA1) commonly present with a
complex, predominantly dystonic movement disorder (MD) following acute or insidious onset …

Use of guidelines improves the neurological outcome in glutaric aciduria type I

J Heringer, SPN Boy, R Ensenauer… - Annals of …, 2010‏ - Wiley Online Library
Objective: To evaluate the effect of treatment according to current evidence‐based
recommendations on the neurological outcome of patients with glutaric aciduria type I (GA …