Management of Acute and Chronic Complications of Lysosomal Storage Diseases in Children and Adults: Current Practice and Future Opportunities

This reprint, entitled “Management of Acute and Chronic Complications of Lysosomal Storage Diseases in Children and Adults: Current Practice and Future Opportunities”, covers broad aspects of this group of rare diseases. The first theme includes articles on the management of pain, corneal clouding,...

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Bibliographic Details
Format:	Online
Language:	English
Published:	MDPI - Multidisciplinary Digital Publishing Institute 2024
Subjects:	Mucopolysaccharidosis corneal clouding penetrating keratoplasty deep anterior lamellar keratoplasty mucopolysaccharidoses airway obstruction management Fabry disease attention Attention Deficit/Hyperactivity cognition late-onset Pompe disease gastrointestinal smooth muscles PROMIS–GI symptom scales GAAKO mice glycogen storage disorder translational research patient-reported outcomes measures mucopolysaccharidosis MPS disease manifestations symptoms morbidity spine disease anaesthesia perioperative complications surgery pompe disease speech voice dysphonia acoustic auditory-perceptual GRBAS respiratory Pompe disease gene panel sequencing alpha-glucosidase GAA dried-blood spots fabry disease neuropathic pain depression Fabry atherosclerosis ischaemia perfusion angina Sanfilippo syndrome mortality Niemann–Pick C neurofilament light biomarkers neurological disease glycogen storage disease type II enzyme replacement therapy COVID-19 inherited metabolic disorders rare autoinflammatory diseases health care providers Gaucher disease NIHF perinatal-lethal Gaucher disease PLGD ichthyosis GBA gene airway management radiology hyoid bone chin intubation intratracheal lysosomal storage disease (LSD) Niemann–Pick type A Niemann–Pick type B acid sphingomyelinase sphingolipid deposition trehalose mucolipidosis type II ML II I-cell disease hyperparathyroidism mucolipidosis ML MLII transition service adult metabolic medicine collaboration challenges inherited metabolic diseases healthcare systems thema EDItEUR::M Medicine and Nursing thema EDItEUR::M Medicine and Nursing::MK Medical specialties, branches of medicine::MKJ Neurology and clinical neurophysiology
Online Access:	ONIX_20240108_9783036597485_81
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Management of Acute and Chronic Complications of Lysosomal Storage Diseases in Children and Adults: Current Practice and Future Opportunities

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