Treatment with Enzyme Replacement Therapy: Causes & Reasons

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Possible Causes for Treatment with Enzyme Replacement Therapy

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]

Life expectancy is typically ten years of age but with treatment may be increased. [nxgenmdx.com]

Mucopolysaccharidosis Type 1 H/S

[ 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]

The treatment has had mixed results. Other treatments depend on the organs that are affected. [en.wikibooks.org]

Fabry Disease

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]

Specific treatment with enzyme replacement therapy (ERT) has been shown to be effective when treatment is started early enough, before irreversible changes can occur. [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]

Available treatments include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). [gaucherdisease.org]

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]

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]

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]

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]

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]

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]

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

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

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

Treatment options include enzyme replacement therapy and hematopoietic stem cell transplantation. [jcor.in]

Although the precise enzyme defect is not known, it is thought to be a sulfatase, either for N-acetyl galactosamine-6-sulfate or for galactose-6-sulfate. [jcor.in]

Perinatal Lethal Gaucher Disease

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]

What is the treatment for Gaucher disease? Enzyme replacement therapy is now available as an effective treatment for individuals who have symptoms from Gaucher disease. [genome.gov]

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 7

Fox, JE, Volpe, L, Bullaro, J, Kakkis, ED, Sly, WS First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient. Mol. [rarediseases.org]

First human treatment with investigational rhGUS enzyme replacement therapy in an advanced stage MPS VII patient. Mol Genet Metab. 2015 Feb. 114 (2):203-8. [Medline]. [emedicine.medscape.com]

Multiple assays of other specific treatments are being performed in animal models: allogenic bone marrow transplantation, gene therapy and enzyme replacement therapy (with [orpha.net]

Niemann-Pick Disease Type B

Treatment Treatment Options: Enzyme replacement therapy trials are underway. [disorders.eyes.arizona.edu]

Enzyme replacement therapy, bone marrow transplantation, and gene therapy are being studied for type B disease. [medindia.net]

Researchers continue to study possible treatments, including enzyme replacement therapy. [contact.org.uk]

Hypophosphatasia

Treatment with enzyme replacement therapy has been reported to improve outcomes in perinatal, infantile, and childhood forms of HPP. [hindawi.com]

We describe a full-term boy diagnosed with perinatal HPP after birth, showing dramatic improvement after treatment with Asfotase Alfa, an enzyme-replacement therapy (ERT) [ncbi.nlm.nih.gov]

CONCLUSIONS: ENB-0040, an enzyme-replacement therapy, was associated with improved findings on skeletal radiographs and improved pulmonary and physical function in infants [ncbi.nlm.nih.gov]

GM2-Gangliosidosis Types B - B1 - Ab

In particular, methods are provided for the treatment of Tay-Sachs disease, Sandhoff Disease, and GM1-gangliosidosis using enzyme replacement therapy. [patentscope.wipo.int]

[…] of lysosomal storage disorders using gene replacement therapy. [patentscope.wipo.int]

FR) MÉTHODES DE TRAITEMENT DE LA MALADIE DE TAY-SACHS, DE LA MALADIE DE SANDHOFF, ET DES GANGLIOSIDOSES À GM1 Abrégé (EN) The present disclosure provides methods for the treatment [patentscope.wipo.int]

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]

Gaucher Disease Type 3

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]

AUG 2000; 26 (4) : 285-290 Beutler-E Commentary: Dosage-response in the treatment of Gaucher disease by enzyme replacement therapy BLOOD-CELLS-MOLECULES-AND-DISEASES. [malattierare.regione.veneto.it]

Adenosine Deaminase Deficiency

Although hematopoietic stem cell transplantation remains the mainstay of treatment for ADA-deficient SCID, 2 other treatment options are available, namely enzyme replacement [ncbi.nlm.nih.gov]

Traditional treatment options, such as enzyme replacement therapy, are of limited efficacy, but bone marrow transplant from a compatible donor leads to a better response. [news-medical.net]

Treatment options comprise enzyme replacement therapy, hematopoeitic stem cell transplantation, and gene therapy. [symptoma.com]

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]

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]

[…] 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]

Disorder of Peroxisomal Alpha- Beta- and Omega-Oxidation

Supportive care, enzyme replacement, bone marrow or stem cell transplantation, experimental gene therapy Increased adenosine deaminase OMIN: 102730 Synonyms: ADA deficiency [extern.synlab.com]

[…] changes, recurrent infections, severe combined immunodeﬁciency, B-cell lymphoma, hemolytic anemia, idiopathic thrombocytopenia, hepatosplenomegaly, mesangial sclerosis; Treatment [extern.synlab.com]

Niemann-Pick Disease

Treatment Treatment Options: Enzyme replacement therapy trials are underway. [disorders.eyes.arizona.edu]

However enzyme replacement therapy, bone marrow and liver transplants may be effective in delaying the onset of NPB. [eurordis.org]

Enzyme replacement therapy delivers the missing enzyme through a vein (IV). Substrate reduction therapy reduces the substance that is building up in the cells. [webmd.com]

Neuronal Ceroid Lipofuscinosis Type 1

These therapies include gene therapy, enzyme replacement therapy, hematopoietic stem cell transplantation, and several medications. [rarediseases.org]

Investigational Therapies There are several different therapies under investigation for the treatment of neuronal ceroid lipofuscinosis including classic infantile CLN1 disease [rarediseases.org]

Exocrine Pancreatic Deficiency

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

It is important to understand this clinical entity and the group of patients requiring treatment with enzyme replacement therapy. [ispub.com]

Enzyme Therapy The most effective treatment for PEI is pancreatic enzyme replacement therapy (PERT), which involves taking a medication called pancrelipase (Cotazym®, Creon [badgut.org]