[PDF] Top 20 Enzyme replacement therapy: efficacy and limitations
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Enzyme replacement therapy: efficacy and limitations
... clinical efficacy of idursulphase treatment. Over 18 months, therapy with atumumab, bortezomib, methotrexate, short-term dexamethasone, and IVIG resulted in a significant reduction in neutraliz- ing ... See full document
10
Clinical efficacy of Enzyme Replacement Therapy in paediatric Hunter patients, an independent study of 3.5 years
... an Enzyme Replacement Therapy (ERT) protocol, employing a recombinant form of ...and efficacy of ERT have been pri- marily evaluated by two randomized trials [9,10] and sub- sequently by an ... See full document
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Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I
... We have shown that a reduction in anti-iduronidase antibod- ies through the induction of rhIDU-specific immune tolerance improves enzyme delivery, uptake into tissues, and clearance of lysosomal storage in the MPS ... See full document
10
Efficacy and safety of enzyme-replacement-therapy with agalsidase alfa in 36 treatment-naïve Fabry disease patients
... All patients underwent measurement of vital signs (blood pressure (BP), pulse rate, and body temperature), and were examined for the incidence of infusion related reactions (rashes, chills, nasal discharge, etc.) both ... See full document
9
Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease
... antiproteinuric therapy with angiotensin-converting enzyme inhibitors or angiotensin receptor blockers throughout the study might be useful for evaluating proteinuria and albuminuria, the variability found ... See full document
10
Enzyme replacement therapy in a feline model of Maroteaux Lamy syndrome
... tolerated enzyme administration with minimal side effects, indicating the potential viability of long term ERT at least in the feline model for MPS ...after enzyme administration indicates antibody binding ... See full document
11
Enzyme-Replacement Therapy With Agalsidase Alfa in Children With Fabry Disease
... though the results presented here demonstrate that bi- weekly infusions of agalsidase alfa in pediatric patients with FD are safe, it is not known whether the otherwise encouraging preliminary results of these multiple ... See full document
11
An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy
... in enzyme replacement therapy (ERT)-naïve children with Fabry ...secondary efficacy endpoints: mean (95% CI) change from baseline at week 55 in LVMI, ... See full document
11
Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports
... human enzyme β-glu- cocerebrosidase, is considered as first-line therapy, and has been shown to improve hematologic parameters, organo- megaly and, to a lesser degree, bone involvement ...the ... See full document
6
Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report
... The efficacy of miglustat has previously been demon- strated over the short term and long term in patients with GD regarding both reduced visceral involvement (sustained reductions in both liver and spleen ... See full document
6
Enzyme replacement therapy for pancreatic insufficiency: present and future
... Pancreatic enzyme replacement therapy is currently the mainstay of treatment for nutrient malabsorption secondary to pancreatic ...demonstrate efficacy in reducing steatorrhea and fat ... See full document
19
Role of elosulfase alfa in mucopolysaccharidosis IVA
... Abstract: Mucopolysaccharidosis type IVA (MPS IVA or Morquio A) is an autosomal recessive lysosomal storage disease which results in a striking skeletal phenotype, but does not negatively impact the intellect of the ... See full document
8
Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease
... An important lesson is to be learned from the limita- tions cited for our study. Sufficient and reliable data on the natural course of a disease are essential not only to assess the effectiveness of future treatments, ... See full document
12
Combination therapy in a patient with chronic neuronopathic Gaucher disease: a case report
... miglustat therapy, the patient also followed specific diet- ary modifications, avoiding high intake of carbohydrate- containing food in single meals, especially foods high in disaccharides, such as sucrose and ... See full document
5
Adenosine deaminase deficiency: a review
... outcome of 106 patients with ADA-deficient SCID fol- lowing HSCT, earlier HSCT was associated with a bet- ter overall survival but this did not reach statistical significance, possibly due to smaller patient numbers in ... See full document
7
Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome
... We acknowledge the limitations of our study, includ- ing cohort size, especially amongst the ERT group, and retrospective nature of data collection. Full multichannel polysomnography was not available in a ... See full document
13
A systematic review of new advances in the management of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): focus on galsulfase
... Enzyme therapy for MPS VI using galsulfase has been shown to result in clinical improvements in endurance (as measured by the 12MWT and 3MSC test) along with a reduction in urinary GAG levels, using the ... See full document
10
The new frame for Mucopolysaccharidoses
... different enzyme deficiencies (Table ...residual enzyme activity in each MPS patient which is related to the specific genetic dysfunction (for details, see the papers by Galimberti et ... See full document
6
Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry
... home therapy for at least 12 months re- ported that five IRRs occurred in two patients ...receiving therapy in a clinical setting to receiving infusions at home ... See full document
9
Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective
... Metachromatic leukodystrophy (MLD) is an autosomal recessively inherited metabolic disease characterized by deficient activity of the lysosomal enzyme arylsulfatase A. Its deficiency results in accumulation of ... See full document
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