Short and long-term acceptability and efficacy of extended-release cornstarch in the hepatic glycogen storage diseases: results from the Glyde study.

Autor: Weinstein DA; Glycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA. weinsteingsd@gmail.com.; School of Medicine, Department of Pediatrics, University of Connecticut, Farmington, CT, USA. weinsteingsd@gmail.com., Jackson RJ; Liverpool Clinical Trials Centre, University of LiverpoolUK, Liverpool, UK., Brennan EA; Vitaflo International Ltd, 182 Sefton Street, Liverpool, UK., Williams M; Glycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA., Davison JE; Metabolic Medicine, Great Ormond Street Hospital, London, UK., Boer F; Division of Metabolic Diseases, Beatrix Children's Hospital, University Medical Center Groningen, Groningen, The Netherlands., Derks T; Division of Metabolic Diseases, Beatrix Children's Hospital, University Medical Center Groningen, Groningen, The Netherlands., Ellerton C; Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK., Faragher B; Liverpool School of Tropical Medicine, Pembroke Place, Liverpool, UK., Gribben J; Evelina London Childrens Hospital, Westminster Bridge Road, London, UK., Labrune P; Centre de Référence des Maladies héréditaires du Métabolisme Hépatique, APHP, Hôpitaux Universitaires Paris-Saclay, Hôpital Antoine Béclère, Clamart, and Paris-Saclay University, Paris, France., McKittrick KM; Vitaflo International Ltd, 182 Sefton Street, Liverpool, UK., Murphy E; Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK., Ross KM; Glycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA., Steuerwald U; National Hospital of the Faroe Islands, Medical Center, Tórshavn, Faroe Islands., Voillot C; Centre de Référence des Maladies héréditaires du Métabolisme Hépatique, APHP, Hôpitaux Universitaires Paris-Saclay, Hôpital Antoine Béclère, Clamart, and Paris-Saclay University, Paris, France., Woodward A; Evelina London Childrens Hospital, Westminster Bridge Road, London, UK., Mundy HR; Evelina London Childrens Hospital, Westminster Bridge Road, London, UK.

Publikováno v: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Jul 09; Vol. 19 (1), pp. 258. Date of Electronic Publication: 2024 Jul 09.

The regulation of growth in glycogen storage disease type 1

Autor: Mundy, HR, Hindmarsh, PC, Matthews, DR, Leonard, JV, Lee, PJ

OBJECTIVE: To study endocrine and metabolic variables that affect growth in patients with glycogen storage disease type 1 (GSD-1) receiving standard dietary therapy. DESIGN: Observational study. PATIENTS AND MEASUREMENTS: Thirty-eight patients with G

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::741cb96bfe5572d5dbbfab7a123dd162
https://doi.org/10.1046/j.1365-2265.2003.01717.x

Safety and tolerability of sodium-glucose cotransporter-2 inhibitors in children and young adults: a systematic review and meta-analysis.

Autor: Nagendra, Lakshmi¹, Dutta, Deep² deepdutta2000@yahoo.com, Girijashankar, Harish Bukkasagar³, Khandelwal, Deepak⁴, Lathia, Tejal⁵, Sharma, Meha⁶

Publikováno v: Annals of Pediatric Endocrinology & Metabolism. Apr2024, Vol. 29 Issue 2, p82-89. 8p.

Reduction in bone mineral density in glycogenosis type III may be due to a mixed muscle and bone deficit.

Autor: Mundy HR; Metabolic Unit, Great Ormond Street Hospital, University College, London, UK., Williams JE, Lee PJ, Fewtrell MS

Publikováno v: Journal of inherited metabolic disease [J Inherit Metab Dis] 2008 Jun; Vol. 31 (3), pp. 418-23. Date of Electronic Publication: 2008 Apr 04.

The effect of L-alanine therapy in a patient with adult onset glycogen storage disease type II.

Autor: Mundy HR; Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK., Williams JE, Cousins AJ, Lee PJ

Publikováno v: Journal of inherited metabolic disease [J Inherit Metab Dis] 2006 Feb; Vol. 29 (1), pp. 226-9.