Treatment with Enzyme Replacement Therapy: Causes & Reasons

Possible Causes for Treatment with Enzyme Replacement Therapy

There is no cure, but treatments such as enzyme replacement therapies can help make MPS VI a more manageable disease. In June 1, 2005, the U.S. [mpssociety.org]

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]

Mucopolysaccharidosis Type 1 H/S

Treatment with enzyme replacement therapy is available. [logan.testcatalog.org]

[ edit ] Enzyme replacement therapy adds a working form of the missing enzyme to the body. [en.wikibooks.org]

Differential diagnosis Other forms of MPS I ; Hunter Syndrome ; other mucopolysaccharidoses Treatment Enzyme replacement therapy with iduronidase ; surgery Prognosis Life [en.wikipedia.org]

Fabry Disease

[…] with enzyme replacement therapy, Nephrology Dialysis Transplantation, Volume 23, Issue 5, May 2008, Pages 1600–1607, https://doi.org/10.1093/ndt/gfm848 Download citation [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]

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]

enzyme replacement therapy. [ncbi.nlm.nih.gov]

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]

Mucopolysaccharidosis Type 2

There is no cure, but treatments like enzyme replacement therapies can help make the disease more manageable. [mpssociety.org]

Enzyme replacement therapy with recombinant iduronate-2-sulphatase (idursulfase), however, has now been introduced. [ingentaconnect.com]

There is no cure but treatments such as enzyme replacement therapies can help make the disease more manageable. [mpssociety.ca]

Mucopolysaccharidosis Type 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]

There is no cure, but treatments such as enzyme replacement therapies can help make MPS VI a more manageable disease. In June 1, 2005, the U.S. [mpssociety.org]

Food and Drug Administration (FDA) granted marketing approval for Naglazyme (galsulfase), the first enzyme replacement therapy approved for the treatment of MPS VI. [mpssociety.org]

Mucopolysaccharidosis Type 1

Available treatment includes enzyme replacement therapy, the incorporation of the missing enzyme into the body through the veins. [nxgenmdx.com]

Treatment with enzyme replacement therapy is available. [neurology.testcatalog.org]

The enzyme replacement therapy should be applied once a week as a life-long treatment. [scinapse.io]

Mucopolysaccharidosis Type 1 H

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]

Pompe Disease

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]

Treatment and prognosis Although no definitive cure exists, treatment with enzyme replacement therapy has been shown to prolong life. [radiopaedia.org]

MRI appearances of skeletal muscle can also be used to assess progress upon commencement of treatment. [radiopaedia.org]

Gaucher Disease Type 2

Enzyme replacement therapy Enzyme replacement therapy is the treatment for types 1 and 3 Gaucher’s disease. [healthline.com]

Enzyme replacement therapy with imiglucerase (Cerezyme), a recombinant version of β glucocerebrosidase, for patients with types I or III ( Curr Opin Pediatr 2007;19:628 ) [pathologyoutlines.com]

Newer treatments include enzyme replacement therapy. [encyclopedia.com]

Late-Onset Pompe Disease

[…] for Pompe disease until enzyme replacement therapy (ERT) was first attempted in 1973. [ncbi.nlm.nih.gov]

Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease. Lab Invest 2006; 86: 1208-20. 6. Hirschhorn R, Reuser A. [medicinabuenosaires.com]

Current treatment consists of enzyme replacement therapy (ERT), which is reimbursed in Belgium since 2006 [9, 10], and supportive therapies such as (non-)invasive ventilation [ojrd.biomedcentral.com]

Mucopolysaccharidosis Type 4A

Management and treatment Before enzyme replacement therapy (ERT) with galsulfase (Naglazyme®), clinical management was limited to supportive care and hematopoietic stem cell [rarediseases.info.nih.gov]

In 2014, Vimizin was approved by the FDA as a enzyme replacement therapy for MPS IVA. There is no treatment for MPS IVB. [mpssociety.org]

Enzyme replacement for Gaucher’s disease. https://www.gaucherdisease.org/gaucher-diagnosis-treatment/treatment/enzyme-replacement-therapy/ National MPS Society. [smartpharmtx.com]

Mucopolysaccharidosis Type 3

Outcome, Enzyme Replacement Therapy, GALNS Protein, Human, Galsulfase, Idursulfase pp.247-252 発行日 2016年3月1日 Published Date 2016/3/1 DOI https://doi.org/10.18888/J00639.2016153583 [webview.isho.jp]

Treatment with enzyme replacement therapy has allowed the missing enzyme to be replaced with the aim to reduce the effects of the disease and prevent it progressing. [cochrane.org]

Enzyme replacement therapy (ERT) has not been shown to be effective in MPS III. [mpssociety.org]

Perinatal Lethal Gaucher Disease

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]

Pancreatic Insufficiency

Pancreatic enzyme replacement therapy (PERT) is the mainstay of treatment, whereby enteric-coated mini-microspheres are taken with every meal, in progressive doses based on [ncbi.nlm.nih.gov]

Pancreatic enzyme replacement therapies Your doctor may start you on a prescription treatment called pancreatic enzyme replacement therapy, or PERT. [pancreasfoundation.org]

Treatment Pancreatic enzyme replacement therapy can treat EPI. Pancreatic enzyme replacement therapy (PERT) is the standard treatment for EPI. [medicalnewstoday.com]

Mucopolysaccharidosis Type 1S

Continued Treatment Enzyme replacement therapy (ERT) uses a drug called laronidase (Aldurazyme), a man-made version of the missing protein. [webmd.com]

To date, there are no published reports of TB treatment in MPS IS patients receiving enzyme replacement therapy (ERT) and as such it is not known whether both conditions can [frontiersin.org]

Mucopolysaccharidosis Type 4

In 2014, Vimizin was approved by the FDA as a enzyme replacement therapy for MPS IVA. There is no treatment for MPS IVB. [mpssociety.org]

Some related diseases already have some experimental treatments such as gene therapy, enzyme replacement, and chaperone therapy. [morquiob.org]

Enzyme replacement therapy with laronidase aims to replace the missing enzyme; however, given its high cost, it is essential to assess how effective and safe this treatment [cochrane.org]

Congenital Disorder of Glycosylation Type 1X

Researchers are studying enzyme replacement therapy for the treatment of CDG. [rarediseases.org]

Enzyme replacement therapy involves replacing the missing enzyme in individuals who are deficient or lack the particular enzyme in question. [rarediseases.org]

[…] component of a consortium of 10 clinical institutions in the US (Frontiers of Congenital Disorders of Glycosylation Consortium) examining the natural history, biomarkers and therapy [rarediseases.org]

Gaucher Disease Type 3

Miglustat is in a class of medications called enzyme inhibitors. ❓ How is Gaucher disease treated? Treatment Enzyme replacement therapy. [fi.health-root.com]

The cause of Gaucher disease III is the accumulation of a fatty material called glucocerebroside (also known as glucosylceramide) Treatment includes enzyme replacement therapy [gii.co.jp]

The treatment of Gaucher disease at present is mainly by enzyme replacement therapy which is expensive. [cochranelibrary.com]

Myoadenylate Deaminase Deficiency

development of enzyme replacement therapies; and development of gene therapies. [mda.org]

[…] the role of exercise and diet in metabolic diseases; development of animal models of metabolic diseases, both to improve understanding of the diseases and to test possible treatments [mda.org]

Gaucher Disease Type 1

Treatment with enzyme replacement therapy has been available for the past two decades but, although effective, enzyme replacement therapy can be delivered only by intravenous [ncbi.nlm.nih.gov]

Early treatment with enzyme replacement therapy (ERT) may prevent disease complications. [ncbi.nlm.nih.gov]

[…] of enzyme replacement therapy as well as longer-term follow-up. [ncbi.nlm.nih.gov]

Mucopolysaccharidosis Type 4C

ERT, enzyme replacement therapy; MPS I, mucopolysaccharidosis type I. [adc.bmj.com]

Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. [ojrd.biomedcentral.com]

Enzyme replacement therapy (ERT) for MPS IVA, for which clinical trials are currently underway (http://clinicaltrials.gov), may be a treatment option in the future. [link.springer.com]

Molybdenum Cofactor Deficiency Type A

Intrathecal enzyme replacement therapy is being tried in some mucopolysaccharidoses with cognitive involvement, in an attempt to halt neurodegeneration. [pesquisa.bvsalud.org]

In recent years the use of enzyme replacement therapy has become more widely extended to treat mucopolysaccharidosis type IVA (Morquio A), mucopolysaccharidosis type VII [pesquisa.bvsalud.org]

Today, the possibilities of treatment for lysosomal diseases have improved. [pesquisa.bvsalud.org]

Imiglucerase

Enzyme replacement (glucocerebrosidase) Comment Enzyme replacement therapy product Treatment of Gaucher disease Target GBA* [HSA_VAR: 2629v1 ] [HSA: 2629 ] [KO: K01201 ] [kegg.jp]

However, a report from a panel of clinicians that treat Gaucher's disease indicated that treatment with enzyme replacement therapy during pregnancy led to a reduced risk of [pdr.net]

Enzyme replacement (glucocerebrosidase) Disease Gaucher disease [DS:H00126] Comment Enzyme replacement therapy product Target GBA* [HSA_VAR:2629v1] [HSA:2629] [KO:K01201 [genome.jp]

Alpha-Mannosidosis

Bone marrow or stem cell transplantation is an effective treatment. Enzyme replacement therapy is currently under investigation. [nxgenmdx.com]

Currently there are two ideas for potential treatments: Enzyme replacement therapy: The missing enzyme is artificially manufactured and then intravenously introduced into [lukechaplin.wordpress.com]

Enzyme Replacement Therapy Segment Serves Maximum Demand Treatment-wise, the report segments the global alpha mannosidosis market into bone marrow transplant and enzyme replacement [transparencymarketresearch.com]

Mucopolysaccharidosis Type 3B

Enzyme replacement therapy (ERT) has not been shown to be effective in MPS III. [mpssociety.org]

(laronidase), an FDA-approved enzyme replacement therapy (ERT). [checkrare.com]

(by physical and occupational therapists) and supportive care is required Currently, enzyme replacement therapy and bone marrow transplant have not been found to be very [dovemed.com]

Alglucerase

Enzyme replacement (glucocerebrosidase) Comment Enzyme replacement therapy product Treatment of Gaucher disease Target GBA* [HSA_VAR:2629v1] [HSA:2629] [KO:K01201] Brite [genome.jp]

Home treatment with intravenous enzyme replacement therapy for Gaucher disease: an international collaborative study of 33 patients. Blood 1993;82(4):1107–9. [link.springer.com]

FYKQPMFYHL GHFSKFIPEG SQRVGLVASQ KNDLDAVALM HPDGSAVVVV LNRSSKDVPL TIKDPAVGFL ETISPGYSIH TYLWRRQ Type Peptide Remark ATC code: A16AB01 Efficacy Lysosomal storage disease treatment [genome.jp]

Congenital Disorder of Glycosylation

Researchers are studying enzyme replacement therapy for the treatment of CDG. [rarediseases.org]

Enzyme replacement therapy involves replacing the missing enzyme in individuals who are deficient or lack the particular enzyme in question. [rarediseases.org]

Storage Disease

We have a dedicated Infusion Center where patients with Gaucher, Fabry, Pompe, MPS, LALD, and Batten disease can receive regular treatment with enzyme replacement therapy. [mountsinai.org]

[…] assay to confirm specific enzyme deficiency (definitive test) Treatment Symptomatic and palliative treatment Enzyme replacement therapy Bone marrow transplantation Hunters [amboss.com]

LSD Treatment and Clinics at CHOP Treatment for LSDs includes enzyme replacement therapy and bone marrow transplants to maximize patient outcomes. [chop.edu]

Perinatal Hypophosphatasia

Asfotase alfa appears to be a potential enzyme replacement therapy in patients with life-threatening HPP. [osteologia.org.ar]

However, since 2015 treatment has been available in the form of Asfotase alpha enzyme replacement therapy 8. [radiopaedia.org]

REVIEW ARTICLES Whyte MP: Hypophosphatasia: enzyme replacement therapy bring new opportunities and new challenges (perspective). [rarediseases.org]