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PubMed Journals Articles About "Catalyst Pharmaceuticals Firdapse Garners Approval Lambert Eaton Myasthenic" RSS

20:56 EDT 20th September 2019 | BioPortfolio

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Showing "Catalyst Pharmaceuticals Firdapse garners approval Lambert Eaton myasthenic" PubMed Articles 1–25 of 1,800+

Lambert-Eaton myasthenic syndrome and merkel cell carcinoma.

Lambert-Eaton myasthenic syndrome (LEMS) is an antibody-mediated disorder of the neuromuscular junction that is most commonly diagnosed in association with small cell lung carcinoma (SCLC). Small cell lung carcinoma is histologically similar to the aggressive cutaneous neuroendocrine malignancy Merkel cell carcinoma (MCC). We provide a full report and longitudinal clinical follow-up of a case of LEMS occurring with MCC. We also review the literature on paraneoplastic syndromes associated with MCC and other ...


Validation of the Triple Timed Up-and-Go Test in Lambert-Eaton Myasthenia.

There are no validated, practical and quantitative measures of disease severity in Lambert-Eaton Myasthenia (LEM).

Trouble at the junction: when myopathy and myasthenia overlap.

Although myopathies and neuromuscular junction disorders are typically distinct, their coexistence has been reported in several inherited and acquired conditions. Affected individuals have variable clinical phenotypes but typically display both a decrement on repetitive nerve stimulation and myopathic findings on muscle biopsy. Inherited causes include myopathies related to mutations in BIN1, DES, DNM2, GMPPB, MTM1 or PLEC and congenital myasthenic syndromes due to mutations in ALG2, ALG14, COL13A1, DOK7, D...


Pharmacokinetics and Tissue Distribution of 3,4-Diaminopyridine in Rats.

Lambert-Eaton myasthenic syndrome (LEMS) is characterized by muscle weakness, amyotrophy, easy fatigability, and depressed tendon reflexes. 3,4-Diaminopyridine (3,4-DAP) is the recommended therapy for treatment of LEMS. However, estimations of 3,4-DAP pharmacokinetics in human and animals, such as rats, are rarely reported because 3,4-DAP is an orphan drug for treatment of a very rare disease (LEMS). In particular, little is known about its tissue distribution. Therefore, we studied the pharmacokinetics of ...

Congenital myasthenic syndrome due to rapsyn deficiency: A case report with a new mutation and compound heterozygosity.

The congenital myasthenic syndromes are a heterogeneous group of genetic disorders characterized by an abnormal synaptic transmission in the neuromuscular plate.

Rational Design of an Organocatalyst for Peptide Bond Formation.

Amide bonds are ubiquitous in peptides, proteins, pharmaceuticals and polymers. The formation of amide bonds is a relatively straightforward process: amide bonds can be synthesized with relative ease because of the availability of efficient coupling agents. However, there is a substantive need for methods that do not require excess reagents. A catalyst that condenses amino acids could have an important impact by reducing the significant waste generated during peptide synthesis. We describe the rational desi...

Mytilidae as model organisms in the marine ecotoxicology of pharmaceuticals - A review.

Growing production and consumption of pharmaceuticals is a global problem. Due to insufficient data on the concentration and distribution of pharmaceuticals in the marine environment, there are no appropriate legal regulations concerning their emission. In order to understand all aspects of the fate of pharmaceuticals in the marine environment and their effect on marine biota, it is necessary to find the most appropriate model organism for this purpose. This paper presents an overview of the ecotoxicologica...

Occurrence and ranking of pharmaceuticals in the major rivers of China.

Residual pharmaceuticals have received widespread attention worldwide due to their continuous release and potential hazard to the environment. As a result of rapid development and a large population, China has become a country with high production and consumption of pharmaceuticals. This may be the main reason for the high detection frequencies and concentrations of pharmaceuticals in the aquatic environment in China. Rivers represent an important water resource and play an important role in the sustainable...

Volanesorsen: First Global Approval.

Volanesorsen (Waylivra), an antisense oligonucleotide inhibitor of apolipoprotein CIII (apoCIII) mRNA, is being developed by Ionis Pharmaceuticals through its subsidiary company, Akcea Therapeutics, to treat familial chylomicronemia syndrome (FCS), hypertriglyceridemia and familial partial lipodystrophy (FPL). In May 2019, volanesorsen was approved in the EU for the treatment of adult patients with FCS based on positive results from the multinational, phase III APPROACH and COMPASS studies. Other clinical ...

The congenital myasthenic syndromes: expanding genetic and phenotypic spectrums and refining treatment strategies.

Congenital myasthenic syndromes (CMS) are a group of heterogeneous inherited disorders caused by mutations in genes encoding proteins whose function is essential for the integrity of neuromuscular transmission. This review updates the reader on the expanding phenotypic spectrum and suggested improved treatment strategies.

Duration and variation of the ethics approval process in Germany - an example from a non-interventional study with 44 participating centers.

The bureaucratic and administrative burden for obtaining the obligatory Institutional Review Board (IRB) approval, which confirms the ethical integrity of a scientific project, is high. In order to participate in multicenter studies, most participating centers in Germany require a local second approval. We investigated the variation in the time it took the participating centers of a non-interventional multicenter study to obtain this approval.

Risk factors for the exacerbation of myasthenic symptoms after surgical therapy for myasthenia gravis and thymoma.

Extended thymectomy is employed for patients with myasthenia gravis (MG) and/or thymoma with elevated serum antiacetylcholine receptor antibody (AchR) titers. However, MG symptoms occasionally worsen in postthymectomy patients. We explored the risk factors for exacerbation of MG symptoms after surgical therapy for patients with MG and/or thymoma with an elevated AchR titer. We retrospectively analyzed 90 patients suffering from MG and/or thymoma with an elevated AchR titer who underwent thymectomy in our in...

Occurrence and distribution of pharmaceuticals in raw, finished, and drinking water from seven large river basins in China.

The occurrence of pharmaceuticals in the drinking water is a growing concern in China. In this study, we determined the concentration and distribution of 59 selected pharmaceuticals in raw, finished, and tap water samples from 79 drinking water treatment plants (DWTPs) in 15 cities, encompassing seven large river basins in China, using solid-phase extraction pretreatment and ultra-high-performance liquid chromatography-tandem mass spectrometry analysis. Forty-seven pharmaceuticals were detected in raw water...

Remogliflozin Etabonate: First Global Approval.

Remogliflozin, a selective sodium-glucose co-transporter subtype 2 (SGLT2) inhibitor, which is to be administered as remogliflozin etabonate (Remo™, Remozen™), the prodrug for remogliflozin, recently received its first approval as a treatment for type 2 diabetes mellitus (T2DM) in India. This article summarizes the milestones in the development of remogliflozin etabonate leading to this first approval for T2DM.

U.S. Emergency Department Visits Resulting From Nonmedical Use of Pharmaceuticals, 2016.

National data on morbidity from nonmedical use of pharmaceuticals are limited. This study used nationally representative, public health surveillance data to characterize U.S. emergency department visits for acute harms from nonmedical use of pharmaceuticals and to guide prevention efforts.

Therapeutic value and price of the new pharmaceuticals commercialized in Argentina: Are they worth what they cost?

In Argentina, new drugs can be authorized by presenting the drug's certificate of approval in at least one of 15 countries considered to have rigorous health surveillance, without needing to carry out a local evaluation of the efficacy, safety or added therapeutic value of the new product. In this article, we evaluate the new drugs commercialized in Argentina in 2016 using different approaches: their approval by other regulatory agencies, the demonstration of their efficacy in randomized clinical trials, ty...

Publications of William A. Eaton.

Tribute to William A. Eaton.

Colleagues of William A. Eaton.

Autobiography of William A. Eaton.

Magnetite/Iron Foil as an Effective and Nonfiltration Catalyst for Heterogeneous Fenton-like Reactions under Neutral Conditions.

A magnetite/iron foil (MIF) composite was synthesized as a heterogeneous Fenton-like catalyst. The MIF catalyst effectively degraded Rhodamine B under neutral conditions (degradation efficiency = 86%), avoiding the procedure of pH adjustment. The MIF catalyst could be conveniently recycled without filtration, and the advantages of the stability and reusability of a MIF catalyst made it promising in practical wastewater treatment.

Lower-dose prescriptions in the post-marketing situation and the influencing factors thereon.

The dosage of pharmaceuticals is determined through the process of clinical development and approval review based on clinical trial results; however, the information obtained from clinical trials before approval is limited. Some pharmaceutical products are used at doses lower than those approved for post-marketing use. The aim of this study was to reveal the actual state of lower-dose prescriptions for post-marketing clinical use of pharmaceuticals. We investigated the factors related to the deviation based...

Abbreviated Curriculum Vitae of William A. Eaton.

Romosozumab: First Global Approval.

Romosozumab (EVENITY™) is a humanised monoclonal antibody against sclerostin being developed by Amgen and UCB for the treatment of osteoporosis. On the basis of favourable results from several phase III trials in postmenopausal women with osteoporosis, and a single trial in men with osteoporosis, romosozumab is being considered for marketing approval in the US, EU and Canada, and was recently approved for marketing in Japan. This article summarizes the milestones in the development of romosozumab leading ...

MIL-100(Fe) supported Mn-based catalyst and its behavior in Hg removal from flue gas.

A novel magnetic composite catalyst of MnOx loaded on MIL-100(Fe) was prepared for the removal of Hg from flue gas, via incipient wetness impregnation followed with calcination at 300 °C. The MIL-100(Fe) supported catalyst showed greater capacity of Hg adsorption and oxidation than FeO supported catalyst at all test temperatures, showing Hg removal efficiency of 77.4% at 250 °C with high GHSV of 18,000 h. Besides the merit of high BET surface area and developed porous, the ultra-highly dispersed and...


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