PubMed Journals Articles About "Amicus Therapeutics Submit Japanese Drug Application Migalastat Fabry" RSS

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Showing "Amicus Therapeutics Submit Japanese Drug Application Migalastat Fabry" PubMed Articles 1–25 of 18,000+

Generation of Fabry cardiomyopathy model for drug screening using induced pluripotent stem cell-derived cardiomyocytes from a female Fabry patient.

Fabry disease is an X-linked disease caused by mutations in α-galactosidase A (GLA); these mutations result in the accumulation of its substrates, mainly globotriaosylceramide (Gb3). The accumulation of glycosphingolipids induces pathogenic changes in various organs, including the heart, and Fabry cardiomyopathy is the most frequent cause of death in patients with Fabry disease. Existing therapies to treat Fabry disease have limited efficacy, and new approaches to improve the prognosis of patients with Fab...

Characterization of drug-neutralizing antibodies in patients with Fabry disease during infusion.

In patients with Fabry disease, neutralizing anti-drug antibodies were identified as IgG4 isotypes. The determination of antibody titers during infusions indicated that high dosages of enzyme overcome anti-drug antibody titers, necessitating individually tailored dose adjustment.

Fabry Nephropathy: An Evidence-Based Narrative Review.

Fabry disease (FD) is a rare, X-linked disorder caused by mutations in the GLA gene encoding the enzyme α-galactosidase A. Complete or partial deficiency in this enzyme leads to intracellular accumulation of globotriaosylceramide (Gb3) and other glycosphingolipids in many cell types throughout the body, including the kidney. Progressive accumulation of Gb3 in podocytes, endothelial cells, epithelial cells, and tubular cells contribute to the renal symptoms of FD, which manifest as proteinuria and reduced g...

Phenotypic characteristics of the p.Asn215Ser (p.N215S) GLA mutation in male and female patients with Fabry disease: A multicenter Fabry Registry study.

The p.Asn215Ser or p.N215S GLA variant has been associated with late-onset cardiac variant of Fabry disease.

Young gastrointestinal angle: How to prepare and submit a job application.

Effectiveness of plasma lyso-Gb3 as a biomarker for selecting high-risk patients with Fabry disease from multispecialty clinics for genetic analysis.

PurposePlasma globotriaosylsphingosine (lyso-Gb3) is a promising secondary screening biomarker for Fabry disease. Here, we examined its applicability as a primary screening biomarker for classic and late-onset Fabry disease in males and females.MethodsBetween 1 July 2014 and 31 December 2015, we screened 2,360 patients (1,324 males) referred from 169 Japanese specialty clinics (cardiology, nephrology, neurology, and pediatrics), based on clinical symptoms suggestive of Fabry disease. We used the plasma lyso...

Hemizygous Fabry disease associated with membranous nephropathy: A rare case report

Fabry disease may coexist with various glomerular diseases, including IgA nephropathy, focal segmental glomerulosclerosis, etc. In this study, we report a rare case of Fabry disease associated with membranous nephropathy (MN).

Identification of Cha o 3 homolog Cry j 4 from Cryptomeria japonica (Japanese cedar) pollen: Limitation of the present Japanese cedar-specific ASIT.

About one-third of the Japanese population suffers from Japanese cedar pollinosis, which is frequently accompanied by Japanese cypress pollinosis. Recently, a novel major Japanese cypress pollen allergen, Cha o 3, was discovered. However, whether a Cha o 3 homolog is present in Japanese cedar pollen remains to be determined.

Proposed Stages of Myocardial Phenotype Development in Fabry Disease.

The authors sought to explore the Fabry myocardium in relation to storage, age, sex, structure, function, electrocardiogram changes, blood biomarkers, and inflammation/fibrosis.

General Hospital and Personal Use Devices; Reclassification of Sharps Needle Destruction Device. Final order.

The Food and Drug Administration (FDA or the Agency) is issuing a final order to reclassify the needle destruction device, renaming the device to "sharps needle destruction device, a postamendments class III device (regulated under product code MTV), into class II (special controls), subject to premarket notification. FDA is also identifying the special controls that the Agency believes are necessary to provide a reasonable assurance of safety and effectiveness of the device. FDA is finalizing this reclassi...

Drug metabolism and pharmacokinetic strategies for oligonucleotide- and mRNA-based drug development.

Oligonucleotide and modified mRNA therapeutics have great potential to treat diseases that are currently challenging to cure and are expanding into global and chronic disease areas such as cancer and various cardiovascular diseases. Advanced drug delivery systems or ligand-drug conjugates are utilized to achieve 'the right dose to the right target' to benefit efficacy and safety in patients. Chemistry and ADME characteristics distinguish these therapeutics from small molecules. Understanding the scalability...

MHRA: confusion over drug names.

Genital angiokeratoma in a woman with Fabry disease: the dermatologist's role.

Fabry disease is a rare lysosomal storage disorder, inherited in an X-linked manner. It is characterized by the deficiency of the enzyme alpha-galactosidase, leading to a buildup of glycosphingolipids in the cells. Angiokeratoma is one of the cutaneous manifestations of this condition, and it helps making the diagnosis. The typical site involves the genital area in men and lumbosacral, buttocks and trunk region in both sexes. We report a case of genital angiokeratoma in a woman with Fabry disease. The diagn...

Screening Test of Fabry Disease in Patients with Renal Replacement Therapy in the City of Modena.

Fabry disease is a rare genetic lysosomal storage disease, inherited in an X-linked manner, characterized by lysosomal deposition of globotriaosylceramide due to deficient activity of the enzyme α-galactosidase A. Because the prevalence of this genetic disorder is unknown in the Emilia Romagna region, we conducted a screening study to assess the prevalence of Fabry disease in the city of Modena, Italy.

Effects of Cytochrome P450 (CYP) 2C19 genotypes on steady-state plasma concentrations of escitalopram and its desmethyl metabolite in Japanese patients with depression.

Plasma concentrations of the S-enantiomer of citalopram were different between extensive and poor CYP2C19 metabolizers in healthy subjects and depressed patients. However, most studies applied dose-corrected concentrations. Thus, we studied the effects of polymorphisms of the CYP2C19 gene on raw plasma drug concentrations in Japanese patients with depression.

Topical drug therapeutics for neuropathic pain.

Neuropathic pain (NP) is a particularly severe and intractable chronic condition that is not well treated by commonly recommended systemic pharmacological therapies, partly due to dose-limiting side effects or adverse events. The use of topical therapeutics for NP is growing and benefits from the reduced potential for adverse effects, as well as the ability to directly target peripheral pathological processes. Areas covered: The current review defines and describes the limitations of various commonly prescr...

Clinic-Pathologic Features and Renal Outcome of Fabry Disease: Data from a Chinese Cohort.

Fabry disease (FD) with life-threatening complications occurs as a result of organ damage in kidneys, heart, and brain. Only a few studies, especially from Asia, report their long-term outcome.

Genomic Screening of Fabry Disease in Young Stroke Patients: the Taiwan Experience and a Review of the Literature.

Fabry disease is a X-linked disease, and enzyme-based screening methods are not suitable for female patients.

Defining Regional Differences in Drug-Induced Stevens-Johnson Syndrome/Toxic Epidermal Necrolysis: A Tool to Improve Drug Safety?

Effect of Canagliflozin on Urinary Albumin Excretion in Japanese Patients with Type 2 Diabetes Mellitus and Microalbuminuria: A Pilot Study.

Albuminuria characterizes the progression of kidney injury. The effect of canagliflozin on the excretion of microalbumin was assessed for investigating its renoprotective potential in Japanese patients with type 2 diabetes mellitus (T2DM).

Nutritional characteristics of the Japanese diet: A cross-sectional study of the correlation between Japanese Diet Index and nutrient intake among community-based elderly Japanese.

To our knowledge, the overall nutritional quality of the Japanese diet has not been assessed previously. The aim of this study was to investigate the relationship between adhering to a Japanese diet and nutrient intake.

Development of an English-language version of a Japanese iPad application to facilitate collaborative goal setting in rehabilitation: a Delphi study and field test.

This study aimed to investigate the content of an English-language version of a Japanese iPad application designed to facilitate shared decision-making around goal setting in rehabilitation: Aid for Decision-making in Occupational Choice-English (ADOC-E).

α-Galactosidase A-deficient rats accumulate glycosphingolipids and develop cardiorenal phenotypes of Fabry disease.

Fabry disease is an X-linked lysosomal storage disease caused by α-galactosidase A (α-Gal A) deficiency. Kidney and heart failure are frequent complications in adulthood and greatly contribute to patient morbidity and mortality. Because α-Gal A-deficient mouse models do not recapitulate cardiorenal findings observed in patients, a nonmouse model may be beneficial to our understanding of disease pathogenesis. In this study, we evaluated disease processes in a recently generated Fabry rat model. We found t...

Thiourea-Based Inhibitors of the B-Cell Lymphoma 6 (BCL6) BTB Domain via NMR-Based Fragment Screening and Computer-Aided Drug Design.

Protein-protein interactions (PPI) between the transcriptional repressor B-cell lymphoma 6 (BCL6) BTB domain (BCL6BTB) and its co-repressors have emerged as a promising target for anti-cancer therapeutics. However, identification of potent, drug-like inhibitors of the BCL6BTB has remained challenging. Using NMR-based screening of a library of fragment-like small molecules, we have identified a thiourea compound (7CC5) that binds to the BCL6BTB. From this hit, the application of computer-aided drug design (C...

Corrigendum to "Tocotrienls: the unsaturated sidekick shifting new paradigms in vitamin E therapeutics" Drug Discov. Today 22 (December (12)) (2017) 1765-1781.

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