High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease - Tabular View -...
All patients, who will have received enzyme replacement therapy (ERT) for at least 2 years, will be evaluated prior to ... High Protein and Exercise therapy along-with Nocturnal Enteral Intervention: Other: High Protein and Exercise Therapy along- ... High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease. This study has been withdrawn ... Diagnosis of Pompe disease; either by enzyme deficiency of muscle biopsy specimen or skin fibroblast culture, or homozygous or ...https://clinicaltrials.gov/ct2/show/record/NCT01656590
Gene therapy to prevent immune response to enzyme replacement therapy
... Posted October 14th, 2007. by pcampeau & filed under ... Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance. Baodong Sun, Andrew ...https://ommbidblog.com/2007/10/14/gene-therapy-to-prevent-immune-response-to-enzyme-replacement-therapy/
Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease | EON:...
... its Phase 1/2 clinical study evaluating the investigational novel enzyme replacement therapy neoGAA in ... alglucosidase alfa enzyme replacement therapy that has been specifically designed for enhanced receptor targeting and enzyme ... its Phase 1/2 clinical study evaluating the investigational novel enzyme replacement therapy neoGAA in 24 patients with late- ... evaluating the investigational novel enzyme replacement therapy neoGAA in 24 patients with late-onset Pompe disease. The safety ...https://www.enhancedonlinenews.com/news/eon/20160303006609/en
Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy
CBS: Dutch Central Bureau of Statistics; ERT: enzyme replacement therapy; GAA: the gene coding for acid alpha-glucosidase; RHS ... Klinge L, Straub V, Neudorf U, Voit T. Enzyme replacement therapy in classical infantile pompe disease: results of a ten-month ... Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease. Neuromuscul Disord. ... Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy. Deniz ...http://pubmedcentralcanada.ca/pmcc/articles/PMC3135500/
Muscle Response to Enzyme Replacement Therapy in Pompe Disease - Tabular View - ClinicalTrials.gov
Muscle Response to Enzyme Replacement Therapy in Pompe Disease. Official Title Muscle Response to Enzyme Replacement Therapy in ... Muscle Response to Enzyme Replacement Therapy in Pompe Disease. This study is currently recruiting participants. See Contacts ... This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy. ...https://clinicaltrials.gov/ct2/show/record/NCT00713453
Clinical Trials Registry | Internet Stroke Center
... enzyme replacement therapy then they must have been off the therapy for at least 30 days and must have a Day-14 antibody blood ... Subject has been previously treated with Replagal or any other enzyme replacement therapy for Fabry Disease. If the patient has ... reactions with prior enzyme replacement therapy.. Subject has been enrolled in another clinical investigative study in the past ... therapy and its adverse events.. Subjects who in the opinion of the investigator (for whatever reason) are thought to be ...http://www.strokecenter.org/trials/clinicalstudies/alternative-dosing-and-regimen-of-replagal-to-treat-fabry-disease/eligibility
Paper: Reduction in Intravenous Infusion Duration of Enzyme Replacement Therapy to Ten Minutes with Velaglucerase Alfa in Adult...
Safe and effective intravenous enzyme replacement therapy (ERT) has been available for more than 25 years for patients with ... 2101 Reduction in Intravenous Infusion Duration of Enzyme Replacement Therapy to Ten Minutes with Velaglucerase Alfa in Adult ...https://ash.confex.com/ash/2017/webprogram/Paper104058.html
MTM Mice Respond to Modified Myotubularin Protein Injections | Quest Magazine Online
Comparing 3E10Fv-MTM1 to similar enzyme replacement therapies such as Myozyme (an FDA-approved drug used to treat the muscle ... In addition to funding enzyme replacement research, MDA is also supporting studies of gene transfer (gene therapy) for MTM, ... 3E10Fv-MTM1 potentially could be developed into an intravenous medication, as are some other enzyme replacement therapies.) ... myopathy showed improvements in muscle strength and function in response to a myotubularin-based enzyme replacement therapy ...http://quest.mda.org/news/mtm-mice-respond-modified-myotubularin-protein-injections
Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal...
Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal ... Enzyme replacement therapy (ERT) with acid α-glucosidase has become available for Pompe disease; however, the response of ... Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal ... In summary, enhanced CI-MPR expression might improve the efficacy of enzyme replacement therapy in Pompe disease through ...https://scholars.duke.edu/display/pub741907
Safety Study of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy - Full Text...
Safety Study of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy. This study ... conducted to test the safety of AT2101 in patients with type 1 Gaucher disease already receiving enzyme replacement therapy ( ... Gaucher disease is a lysosomal storage disorder resulting from a deficiency in the key enzyme beta-glucocerebrosidase (GCase). ... The enzyme deficiency is caused by genetic mutations, which can result in the production of misfolded GCase. AT2101 is designed ...https://clinicaltrials.gov/ct2/show/NCT00433147
FDA clears Alexion's enzyme replacement therapy Kanuma - PharmaTimes
US regulators have issued a green light for Alexion's long-term enzyme replacement therapy Kanuma, giving patients with the ... US regulators have issued a green light for Alexion's long-term enzyme replacement therapy Kanuma, giving patients with the ... Alexion's enzyme replacement therapy for another ultra-rare condition called paediatric-onset hypophosphatasia. ...http://www.pharmatimes.com/news/fda_clears_alexions_enzyme_replacement_therapy_kanuma_972250
Enzyme Replacement Therapy in Fabry Disease - Full Text View - ClinicalTrials.gov
This study wants to evaluate the long term efficacy of enzyme replacement therapy in patients with Fabry disease and renal ... Evaluation of the Long Term Efficacy of Enzyme Replacement Therapy in Fabry Disease. ... Infusions of the enzyme replacement treatment have been demonstrated to be safe and effective. ... In the classic form (where the enzyme activity is absent) the clinical findings are represented by pain and paresthesias in the ...https://clinicaltrials.gov/show/NCT00149318
Enzyme replacement therapy
Tifft, C., Proud, V., Levy, P., DeMarco, K., Nicely, H. and Turbeville, S. (2009) Enzyme Replacement Therapy in the Home ... Galsulfase, a Food and Drug Administration-approved enzyme replacement therapy for mucopolysaccharidosis VI, is administered ... some physicians are transitioning appropriate patients to home infusion therapy. An online survey was conducted with 3 ... 4 patients with mucopolysaccharidosis VI to better understand the factors motivating the transition to home infusion therapy, ...http://www.ivteam.com/intravenous-literature/enzyme-replacement-therapy/
Enzyme Replacement Therapy for Lysosomal Storage Disorders | Effective Health Care Program
ERT enzyme replacement therapy. GSD II glycogen storage disease type II (Pompe disease). IMD inherited metabolic disorders. LSD ... What FDA-approved enzyme replacement therapy (ERT) products are available for lysosomal storage diseases (LSD)?. *What are the ... Enzyme reconstitution/replacement therapy for lysosomal storage diseases. Curr Opin Pediatr 2007 Dec;19(6):628-35. PMID: ... Diseases with FDA-approved Enzyme Replacement Therapy*. Disease [estimated incidence]. Symptoms. FDA-Approved Product (approval ...https://effectivehealthcare.ahrq.gov/topics/lysosomal-storage-diseases-therapies/research-protocol
Enzyme replacement therapy on hypophosphatasia mouse model | Springer for Research & Development
Enzyme replacement therapy (ERT) for HPP by functional TNSALP is one of the therapeutic options. The C-terminal-anchorless ... Millán JL, Narisawa S, Lemire I et al (2008) Enzyme replacement therapy for murine hypophosphatasia. J Bone Miner Res 23:777-87 ... Bone Mineral Density Enzyme Replacement Therapy Fabry Disease Homozygous Mouse Hypophosphatasia These keywords were added by ... McKee MD, Nakano Y, Masica DL et al (2011) Enzyme replacement therapy prevents dental defects in a model of hypophosphatasia. J ...https://rd.springer.com/article/10.1007/s10545-013-9646-7
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) - Study Results - ClinicalTrials.gov
Biological: Iduronate-2-sulfatase enzyme replacement therapy. Biological: iduronate-2-sulfatase enzyme replacement therapy. ... Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II). The safety and scientific validity of ...https://clinicaltrials.gov/ct2/show/results/NCT00069641
Improvement of Cardiac Function During Enzyme Replacement Therapy in Patients With Fabry Disease | Circulation
Enzyme replacement therapy in Fabry disease: a randomized controlled trial. JAMA. 2001; 285: 2743-2749. ... Recently, a new therapeutic approach of enzyme replacement therapy (ERT) with recombinant α-Gal A was introduced.2-3 First ... Background- Enzyme replacement therapy (ERT) has been shown to enhance microvascular endothelial globotriaosylceramide ... Improvement of Cardiac Function During Enzyme Replacement Therapy in Patients With Fabry Disease. A Prospective Strain Rate ...http://circ.ahajournals.org/content/108/11/1299
Enzyme replacement therapy - Wikipedia
Protein replacement therapy Ries, M (2017). "Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016 ... Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. ... Neufeld EF (2006). "Chapter 10: Enzyme replacement therapy - a brief history". In Mehta A, Beck M, Sunder-Plassmann G. Fabry ... "A 24-Year Enzyme Replacement Therapy in an Adenosine-deaminase-Deficient Patient". Pediatrics. 137 (1): e20152169. doi:10.1542/ ...https://en.wikipedia.org/wiki/Enzyme_replacement_therapy
FDA Approves Elaprase, Enzyme Replacement Therapy For Hunter's Syndrome - BioTech Weblog
FDA Approves Elaprase, Enzyme Replacement Therapy For Hunter's Syndrome 0 By Ruth on July 26, 2006. · Drugs, Vaccines and ... You are at:Home»Drugs, Vaccines and Therapeutics»FDA Approves Elaprase, Enzyme Replacement Therapy For Hunter's Syndrome ... It is given as weekly infusions, a therapy that is expected to be one of the world's most expensive medical treatments, at ... Individuals afflicted with this disorder lack an enzyme called iduronate-2-sulfatase (I2S), and are unable to break down and ...http://www.biotech-weblog.com/fda-approves-elaprase-enzyme-replacement-therapy-for-hunters-syndrome/
Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency - Patent # 8709408 - PatentGenius
The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of ... Beutler et al., 1977, "Enzyme replacement therapy in Gaucher's disease: preliminary clinical trial of a new enzyme preparation ... Pentchev, 1977, "Enzyme replacement therapy in Gaucher's and Fabry's disease", Ann. Clin. Lab. Sci. 7:251-253. cited by ... McGovern et al, 2009, "A Phase 1 Trial of Recombinant Human Acid Sphingomyelinase (rhASM) Enzyme Replacement Therapy in Adults ...http://www.patentgenius.com/patent/8709408.html
Enzyme Replacement Therapy Market - Global Industry Analysis, Size, Share, Growth, Trends and Forecast To 2022 | Medgadget
Enzyme Replacement Therapy Industry Development and Investment Report, 2017-2022 This report studie ... This report studies the global Enzyme Replacement Therapy market, analyzes and researches the Enzyme Replacement Therapy ... Enzyme Replacement Therapy can be split into. Parenteral Route. Oral. Market segment by Application, Enzyme Replacement Therapy ... Global Enzyme Replacement Therapy Market Size, Status and Forecast 2022. 3 Company (Top Players) Profiles. 3.1 Genzyme ...https://www.medgadget.com/2017/03/enzyme-replacement-therapy-market-global-industry-analysis-size-share-growth-trends-and-forecast-to-2022.html
A Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure - Full Text View - ClinicalTrials.gov
A Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (CUPID-2b). The safety and scientific ... Medical therapy as appropriate to the individual subject including oral diuretic, angiotensin-converting enzyme (ACE) inhibitor ... Resynchronization therapy, if clinically indicated according to ACC/AHA/ESC HF guidelines, must have been implanted at least 6 ... Any intravenous (IV) therapy with positive inotropes, vasodilators or diuretics within 30 days prior to screening. ...https://clinicaltrials.gov/show/NCT01643330
Enobia Announces Positive Clinical Results With ENB-0040, a Bone Targeted Enzyme Replacement Therapy for Hypophosphatasia
... a bone targeted enzyme replacement therapy, under investigation for the treatment of hypophosphatasia (HPP). ... As an enzyme replacement therapy designed to specifically target TNSALP to the bones, ENB-0040 may help correct the enzyme ... Enobia Announces Positive Clinical Results With ENB-0040, a Bone Targeted Enzyme Replacement Therapy for Hypophosphatasia. ... 14 Enobia Pharma today announced positive data from a clinical study of ENB-0040, a bone targeted enzyme replacement therapy, ...http://www.medindia.net/health-press-release/Enobia-Announces-Positive-Clinical-Results-With-ENB-0040-a-Bone-Targeted-Enzyme-Replacement-Therapy-for-Hypophosphatasia-44231-1.htm
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome - Tabular...
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome. The safety ... Laronidase Enzyme Replacement Therapy will be performed using laronidase once a week for 12 weeks prior to hematopoietic stem ... Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome. ... MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for ...https://clinicaltrials.gov/ct2/show/record/NCT01572636