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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...
Fabry disease is characterized by deficient expression/activity of α-GalA with consequent lysosomal accumulation in various organs of its substrate Gb3. Despite enzyme replacement therapy, Fabry disease progresses with serious myocardial, cerebral and renal manifestations. Gb3 accumulation may induce oxidative stress (OxSt), production of inflammatory cytokines and reduction of nitric oxide, which may impact on Fabry disease's clinical manifestations.
Pharmacokinetics (PK) in Japanese healthy subjects were simulated for nine compounds using physiologically based pharmacokinetic (PBPK) models parameterized with physicochemical properties, preclinical absorption, distribution, metabolism, and excretion (ADME) data, and clinical PK data from non-Japanese subjects. For each dosing regimen, 100 virtual trials were simulated and predicted/observed ratios for C and AUC were calculated. As qualification criteria, it was pre-specified that over 80% of simulated t...
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).
The authors sought to explore the Fabry myocardium in relation to storage, age, sex, structure, function, electrocardiogram changes, blood biomarkers, and inflammation/fibrosis.
BACE1 is required for the production of β-amyloid peptides and considered a potential treatment target for Alzheimer's disease (AD). To support Japan's participation in the global clinical development program, we characterized the safety, pharmacokinetics, and pharmacodynamics of the BACE1 inhibitor verubecestat (MK-8931) in 24 healthy Japanese adults in a 2-part, single-center, randomized, placebo-controlled phase I trial and compared the results with historic data from non-Japanese subjects. Both single ...
It would often be of interest to know the effect of a drug, compared to control, in people who take the drug. However, different people will likely take drug and control. Then comparing takers of drug to takers of control does not yield a drug effect. Drug effects in drug takers can be estimated, but first they must be carefully defined. This article is protected by copyright. All rights reserved.
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...
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...
One of the major challenges in drug development is the delivery of therapeutics to the central nervous system (CNS). The blood-brain barrier (BBB), which modulates the passage of molecules from the CNS, presents a formidable obstacle that limits brain uptake of therapeutics and, therefore, impedes the treatment of multiple neurological pathologies. Targeted nanocarriers present an excellent opportunity for drug delivery into the brain leveraging on endogenous receptors to transport therapeutics across the B...
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...
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.
Fabry disease is a X-linked disease, and enzyme-based screening methods are not suitable for female patients.
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...
To evaluate the presence of white matter and hemorrhagic lesions in brain MRI of children and adolescents with Fabry disease (FD).
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.
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).
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...
Fabry cardiomyopathy (FC) and light-chain amyloid cardiomyopathy (AL) present with concentric left ventricular (LV) hypertrophy/remodeling and diastolic rather than systolic dysfunction. Direct comparisons are difficult due to rarity and confounded by variability of LV thickness.
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...
Severe Bradyarrhythmia Linked to Left Atrial Dysfunction in Fabry disease - a Cross-Sectional Study: Cross-Sectional Analysis of the Clinical, Electrocardiographic and Echocardiographic Determinants of Bradyarrhythmic Events in Patients with Fabry Disease - the Impact of Left Atrial Function.
Fabry disease (FD) is a lysosomal storage disorder caused by an enzymatic deficiency. Conduction abnormalities and bradyarrhythmias are common and can occur prior to the onset of left ventricular (LV) hypertrophy. We aimed to describe the clinical, electrocardiographic and echocardiographic, including left atrial (LA) function, determinants of bradyarrhythmic events in FD.
Laser light at 578 nm is frequency-stabilized to two independent 30-cm-long Fabry-Pérot cavities. To achieve a thermal-noise-limited cavity length stability, the geometry and support configuration of the Fabry-Pérot cavities are optimized. The fractional frequency instability of each cavity-stabilized laser system is 2 × 10 at 1 s averaging time, approaching to the thermal-noise-induced length instability of the reference cavity. The most probable linewidth of each laser system is about 0.2 Hz, and the l...