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

02:26 EDT 22nd March 2018 | BioPortfolio

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Showing "Amicus Therapeutics Submit Japanese Drug Application Migalastat Fabry" PubMed Articles 1–25 of 17,000+

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.

Proposed Industry Best Practices in Development and Marketing of Medical Foods for the Management of Chronic Conditions and Diseases while Awaiting Regulation.

Ideal therapeutics have low toxicity and can effectively manage condition(s) or disease(s). The Food & Drug Administration (FDA) marketing category of therapeutics called “medical foods” (MFs) meets such a definition. Medical foods have existed in Federal law since passage the Orphan Drug Act in 1988, which created a category of nutritional therapeutics separate from drugs. Unfortunately, MFs are not widely understood by the medical community or utilized in all patients who need them due to lack of a FD...

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...

The pathophysiology of Fabry disease.

Fabry disease is a lysosomal condition with systemic clinical expression, caused by the tissue deposit of globotriaosylceramide, due to a deficit in its degradation. As with most lysosomal diseases, the presence of a mutation in a gene does not explain the pathophysiological disorders shown by patients. We conducted a comprehensive review of the pathogenic mechanisms that occur in 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...

PBMs: Will the Amazon Cometh?

PBM consultant Linda Cahn says that if Amazon gets into the PBM business it could insist that drugmakers simultaneously submit the net discount price for each of their drugs for the subsequent six months. Amazon could then publicize every drug's actual price, by drug and by therapeutic category.

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.

A Novel Missense GLA Mutation (p.G35V) Detected in Hemodialysis Screening Leads to Severe Systemic Manifestations of Fabry Disease in Men and Women.

Fabry disease (FD), an X-linked lysosomal storage disorder, leads to accumulation of globotriaosylceramide. Screening in dialysis patients may identify genetic variants of unknown clinical significance. We aimed to characterize the pathogenicity of a novel GLA gene mutation identified during hemodialysis screening and the histologic findings of early Fabry nephropathy.

MHRA: confusion over drug names.

Plasma lyso-Gb3: a biomarker for monitoring fabry patients during enzyme replacement therapy.

Recently, globotriaosylsphingosine (lyso-Gb3) has attracted interest as a biomarker of Fabry disease. However, little is known regarding its utility for the evaluation of the therapeutic efficacy.

Access to benznidazole for Chagas disease in the United States-Cautious optimism?

Drugs for neglected tropical diseases (NTD) are being excessively priced in the United States. Benznidazole, the first-line drug for Chagas disease, may become approved by the Food and Drug Administration (FDA) and manufactured by a private company in the US, thus placing it at risk of similar pricing. Chagas disease is an NTD caused by Trypanosoma cruzi; it is endemic to Latin America, infecting 8 million individuals. Human migration has changed the epidemiology causing nonendemic countries to face increas...

Synthesis of (3S,4S,5S)-trihydroxylpiperidine derivatives as enzyme stabilizers to improve therapeutic enzyme activity in Fabry patient cell lines.

A series of 3S,4S,5S-trihydroxylated piperidines bearing structural diversity at C-2 or C-6 positions has been synthesized and tested to determine their ability to stabilize the activity of recombinant human α-Galactosidase A (rh-α-Gal A). Hit molecules were identified by rapid inhibitory activity screening, and then further investigated for their ability to protect this enzyme from thermo-induced denaturation and enhance its activity in Fabry patient cell lines. Our study resulted in the identification o...

Transparency and Dermatologic Device Approval by the US Food and Drug Administration.

The US Food and Drug Administration approves Class III medical devices via the premarket approval pathway, often requiring clinical data on safety and efficacy. Manufacturers can submit incremental device changes via supplemental applications, which are not subjected to such vetting measures and can cause understudied changes that lead to drift from a device's original design.

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).

Integrins in wound healing, fibrosis and tumor stroma: High potential targets for therapeutics and drug delivery.

Wound healing is a complex process, which ultimately leads to fibrosis if not repaired well. Pathologically very similar to fibrosis is the tumor stroma, found in several solid tumors which are regarded as wounds that do not heal. Integrins are heterodimeric surface receptors which control various physiological cellular functions. Additionally, integrins also sense ECM-induced extracellular changes during pathological events, leading to cellular responses, which influence ECM remodeling. The purpose and sco...

Improvement in Ocular Bioavailability and Prolonged Delivery of Tobramycin Sulfate Following Topical Ophthalmic Administration of Drug-Loaded Mucoadhesive Microparticles Incorporated in Thermosensitive In Situ Gel.

Conventional topical delivery in hyperacute bacterial conjunctivitis and endophthalmitis is associated with low drug bioavailability due to rapid precorneal clearance. Hence, in the present investigation, an attempt has been made to enhance ocular bioavailability of tobramycin sulfate by formulating drug-loaded microparticles dispersed in thermosensitive in situ gel.

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

Establishment of an Evaluation Method to Detect Drug Distribution in Hair Follicles.

Development of an appropriate method to evaluate drug disposition or targeting ability in hair follicles (HFs) is urgently needed in order to develop useful pharmaceutical products with pharmacological effects in HFs. In the present study, a cyanoacrylate biopsy (CB) method was used to measure drug disposition in HFs using a model hydrophilic drug, caffeine (CAF), and a lipophilic drug, 4-butylresorcinol (BR), in excised porcine skin. As a result, the height of HF replicas and the recovery ratio decreased w...

Pharmacokinetic Phenotyping to Predict Drug-Drug Interactions: Time to Divorce the Hybrid Concept of Simultaneous Mechanistic-Based and Exposure-Based Assessment.

Research Progress of MALDI-TOF-IMS in Biomedicine and Its Application Prospect in Forensic Sciences.

Matrix-assisted laser desorption/ionization time-of-flight imaging mass spectrometry (MALDI-TOF-IMS) can analysis unknown compounds in sections and obtain molecule imaging by scanning biological tissue sections, which has become a powerful tool for the research of biomarker, lipid distribution and drug metabolism, etc. This article reviews the application of this technique in protein identification, clinical application, drug discovery, lipid research and brain injury.

Re: Biomedical Informatics Approaches to Identifying Drug-Drug Interactions: Application to Insulin Secretagogues.

BP targets for people with CVD.

Analysis of mainland Japanese and Okinawan Japanese populations using the precision ID Ancestry Panel.

We typed 165 AIMs in 49 mainland Japanese and 47 Okinawa Japanese using the Precision ID Ancestry Panel (Thermo Fisher Scientific). None of the 165 SNPs showed significant deviation from Hardy-Weinberg equilibrium in the mainland Japanese. One SNP (rs3943253) showed significant deviation from Hardy-Weinberg equilibrium in Okinawa Japanese. Fisher's exact tests showed that the genotype frequencies of 14 loci were significantly different (p

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