Mucopolysaccharidosis
Available treatment includes enzyme replacement therapy, the incorporation of the missing enzyme into the body through the veins.[nxgenmdx.com]
Management and treatment Before enzyme replacement therapy (ERT) with galsulfase (Naglazyme ), clinical management was limited to supportive care and hematopoietic stem cell[orpha.net]
(enzyme replacement therapy).[ncbi.nlm.nih.gov]
Mucopolysaccharidosis 1 H-S
Differential diagnosis Other forms of MPS I ; Hunter Syndrome ; other mucopolysaccharidoses Treatment Enzyme replacement therapy with iduronidase ; surgery Prognosis Life[en.wikipedia.org]
[ edit ] Enzyme replacement therapy adds a working form of the missing enzyme to the body.[en.wikibooks.org]
Structure of dermatan sulfate, one of the molecules that accumulates in the lysosomes of MPS I patients Usual onset Age 3-8 years Causes Deficiency of the alpha-L iduronidase enzyme[en.wikipedia.org]
Gaucher Disease
We present the case of a male child with Gaucher disease who developed massive mesenteric lymph nodes despite otherwise successful treatment with enzyme replacement therapy[ncbi.nlm.nih.gov]
Available treatments include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).[gaucherdisease.org]
Protein-losing enteropathy due to massive mesenteric lymphadenopathy is a rare complication of Gaucher disease which is generally refractory to treatment with enzyme replacement[ncbi.nlm.nih.gov]
Fabry Disease
[…] with enzyme replacement therapy, Nephrology Dialysis Transplantation, Volume 23, Issue 5, May 2008, Pages 1600–1607, Download citation file: Ris (Zotero) EndNote BibTex Medlars[ndt.oxfordjournals.org]
Their detection is important for early diagnosis and institution of treatment with enzyme replacement therapy, which prevents late complications reducing morbidity and mortality[ncbi.nlm.nih.gov]
Enzyme replacement therapy is the current mainstay of treatment.[ncbi.nlm.nih.gov]
Mucopolysaccharidosis 6
Management and treatment Before enzyme replacement therapy (ERT) with galsulfase (Naglazyme ), clinical management was limited to supportive care and hematopoietic stem cell[orpha.net]
Treatment Treatment Options: Enzyme replacement therapy with galsulfase (Naglazyme ) is beneficial in alleviating some of the manifestations of this disease.[disorders.eyes.arizona.edu]
The two standard treatments currently used are enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT).[symptoma.com]
Glycogen Storage Disease Type 2
Presymptomatic patients with subtle objective signs of Pompe disease (and patients symptomatic at diagnosis) should begin treatment with enzyme replacement therapy (ERT) immediately[ncbi.nlm.nih.gov]
., Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy. Orphanet J Rare Dis, 2011. 6: p. 34. 35.[rarediseases.org]
Mucopolysaccharidosis 1H
Available treatment includes enzyme replacement therapy, the incorporation of the missing enzyme into the body through the veins.[nxgenmdx.com]
[ edit ] Enzyme replacement therapy adds a working form of the missing enzyme to the body.[en.wikibooks.org]
Enzyme replacement therapy to replace a working form of the enzyme alpha-L-iduronidase.[syndromepictures.com]
Lactose Intolerance
[…] promise in the advancement of digestive enzyme therapy.[ncbi.nlm.nih.gov]
[…] in the advancement of digestive enzyme therapy.[ncbi.nlm.nih.gov]
Enzyme replacement therapy for primary adult lactase deficiency.[web.archive.org]
Osteoporosis
Patient from Figure 1A after 12 months asfotase alfa enzyme replacement therapy When Enzyme Replacement Therapy is Not Available or Not Typically Used Perinatal types.[ncbi.nlm.nih.gov]
When enzyme replacement therapy is not available or not typically used Perinatal types.[web.archive.org]
Treatment of manifestations : Perinatal (severe) type: limited experience with enzyme replacement therapy (ERT); expectant management and family support.[ncbi.nlm.nih.gov]
Gaucher Disease, Collodion Type
Although enzyme replacement therapy (ERT) is an effective treatment for organomegaly, anemia, thrombocytopenia, and bone crisis, there are still unmet GD needs and co-morbidities[bmcmedgenet.biomedcentral.com]
Current standard of care is enzyme replacement therapy, as described below.[dermatologyadvisor.com]
Enzyme Replacement Therapy for Gaucher Disease.” National Gaucher Foundation, www.gaucherdisease.org/gaucher-diagnosis-treatment/treatment/enzyme-replacement-therapy/.[rarediseases.org]