PubMed Journals Articles About "Akcea Pfizer Announce Licensing Agreement Investigative Antisense Therapy" RSS

03:50 EST 14th November 2019 | BioPortfolio

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Showing "Akcea Pfizer Announce Licensing Agreement investigative antisense therapy" PubMed Articles 1–25 of 14,000+

N-acetyl galactosamine-conjugated antisense drug to APOC3 mRNA, triglycerides and atherogenic lipoprotein levels.

Elevated apolipoprotein C-III (apoC-III) levels are associated with hypertriglyceridaemia and coronary heart disease. AKCEA-APOCIII-LRx is an N-acetyl galactosamine-conjugated antisense oligonucleotide targeted to the liver that selectively inhibits apoC-III protein synthesis.

Error-Driven Retrieval in Agreement Attraction Rarely Leads to Misinterpretation.

Previous work on agreement computation in sentence comprehension motivates a model in which the parser predicts the verb's number and engages in retrieval of the agreement controller only when it detects a mismatch between the prediction and the bottom-up input. It is the error-driven second stage of this process that is prone to similarity-based interference and can result in the illusory licensing of a subject-verb number agreement violation in the presence of a structurally irrelevant noun matching the n...

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 ...

Structure-Function Agreement Is Better Than Commonly Thought in Eyes With Early Glaucoma.

To assess the agreement between structural (optical coherence tomography [OCT]) and functional (visual field [VF]) glaucomatous damage with an automated method and deviation/probability maps, and to compare this method to a metric method.

Aminoglycoside Enhances the Delivery of Antisense Morpholino Oligonucleotides In Vitro and in mdx Mice.

Antisense oligonucleotide (AO) therapy has been the specific treatment for Duchenne muscular dystrophy, with ongoing clinical trials. However, therapeutic applications of AOs remain limited, particularly because of the lack of efficient cellular delivery methods imperative for achieving efficacy. In this study, we investigated a few aminoglycosides (AGs) for their potential to improve the delivery of antisense phosphorodiamidate morpholino oligomer (PMO) both in vitro and in vivo. AGs had lower cytotoxici...

Storm warning: lung disease in systemic juvenile idiopathic arthritis.

Dr. Nigrovic's work was supported by the NIH grants R01 AR065538, AR075906, AR073201 and P30 AR070253, the Fundación Bechara and the Arbuckle Family Fund for Arthritis Research. PAN is the recipient of investigator-initiated research grants from Novartis, Pfizer, Sobi, and Bristol-Myers Squibb; consulting fees from Novartis, Pfizer, Sobi, Quench Bio ($10,000); salary support from the Childhood Arthritis and Rheumatology Research Alliance; and royalties from UpToDate and the American Academy of Pediatrics. ...

Teen driver licensure provisions, licensing, and vehicular fatalities.

Between 1996 and 2015, vehicular fatalities per capita involving 16- to 17-year-old drivers declined by 68.7%. During this same period, states enacted teen driver licensure provisions in an á la carte fashion, now collectively referred to as graduated driver licensing (GDL) programs, that restricted teen driving. While the literature demonstrates that 'good' GDL programs reduce vehicular fatalities, how these reductions occur remains open. In this study, separate GDL provisions and no pass, no drive laws a...

R-Loops Promote Antisense Transcription across the Mammalian Genome.

Widespread antisense long noncoding RNA (lncRNA) overlap with many protein-coding genes in mammals and emanate from gene promoter, enhancer, and termination regions. However, their origin and biological purpose remain unclear. We show that these antisense lncRNA can be generated by R-loops that form when nascent transcript invades the DNA duplex behind elongating RNA polymerase II (Pol II). Biochemically, R-loops act as intrinsic Pol II promoters to induce de novo RNA synthesis. Furthermore, their removal a...

Patent pledges, open IP, or patent pools? Developing taxonomies in the thicket of terminologies.

Recently, a range of organisations, including car and consumer electronics manufacturers, have applied so-called patent pledges. A patent pledge is a publicly announced intervention by patent-owning entities ('pledgers') to out-license active patents to the restricted or unrestricted public free from or bound to certain conditions for a reasonable or no monetary compensation. Despite growing research to better understand this phenomenon, the underlying terminology remains contradictory. We apply an inductiv...

Agreement Between Two Procalcitonin Assays in Hospitalized Children.

Agreement between available procalcitonin (PCT) assays is unclear. We sought to compare concordance between Roche and bioMérieux PCT assays using pediatric samples.

Scientific production and licensing of medical schools in Peru.

The new university law 30220 of 2014 introduced the mandatory institutional licensing of all Peruvian universities by the National Superintendence of Higher University Education (SUNEDU, in Spanish). The first undergraduate program to go through this process will be medicine. The licensing of medical programs is necessary to ensure that the conditions in which the program is taught in Peru are adequate, with a high probability of closing some medical schools. Once a medical school has demonstrated that it m...

Diversity is excellence: Initiatives in the Society for Investigative Dermatology to broaden participation.

In this issue of JID, Shukla et al show that the Society of Investigative Dermatology has improved gender diversity of its awards, lectureships, and its leadership. Additional initiatives that promote participation in the Society based on education, gender, race, sexual orientation, and other types of diversity can improve the quality of investigative dermatology and clinical progress for skin diseases.

Therapy with 2'-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal Protein Reabsorption.

Antisense oligonucleotide therapy has been reported to be associated with renal injury. Here, the mechanism of reversible proteinuria was investigated by combining clinical, pre-clinical, and in vitro data. Urine samples were obtained from Duchenne muscular dystrophy (DMD) patients treated with drisapersen, a modified 2'O-methyl phosphorothioate antisense oligonucleotide (6 mg/kg). Urine and kidney tissue samples were collected from cynomolgus monkeys (Macaca fascicularis) dosed with drisapersen (39 week...

Agreement of the Professional Association of German Anesthesiologists and the Professional Association of German Surgeons for the Organization of Postoperative Pain Therapy for Surgical Patients (revised version 2019).

The treatment of any causal, procedure-specific and/or concomitant acute pain is an essential quality feature in any surgical subspecialty. An interdisciplinary and interprofessional pain therapy aims for an immediate improvement of the patients' quality of life, a reduction of the risk of postoperative morbidity and mortality in the medium term, prevention of any pain chronification in the long run as well as a reduction of hospitalization and sick leave. The "Agreement of the Professional Association of...

Shrink the giant: scale down the citrus tree to a model system to investigate the RNA interference efficiency.

Virus-induced silencing gene technology has been increasingly used; however, a controversy exists among researchers about whether using the sense or antisense orientation of the gene target is more efficient. Herein, instead of using the entire citrus tree, a reduced system consisting of a single leaf, 5 cm of the stem and a few roots was established to fairly compare between the sense or antisense orientation of phytoene desaturase gene (pds) in the Citrus tristeza virus vector, for improved RNAi efficienc...

Nanoparticle Targeting to Scalp Hair Follicles - New Perspectives for a Topical Therapy of Alopecia Areata.

Negative phase III trials announce the need for biomarkers in sarcoma.

Defining a minimal effective serum trough concentration of secukinumab in psoriasis: a step towards personalized therapy.

How to evaluate agreement between quantitative measurements.

When a method comparison study is performed, the aim is to evaluate the agreement of measurements of different methods. We present the Bland-Altman plot with limits of agreement as the correct analysis methodology. We also discuss other scaled and unscaled indices of agreement and commonly used inappropriate approaches.

Change in baseline characteristics over 20 years of adults with growth hormone (GH) deficiency on GH replacement therapy.

Clinical observations over time of adults with growth hormone (GH) deficiency (GHD) have indicated a shift in patient characteristics at diagnosis. The objective was to compare baseline characteristics of patients diagnosed with adult onset GHD naive to GH replacement during three study periods [1994-1999 (P1), 2000-2004 (P2), and 2005-2012 (P3)] using the KIMS (Pfizer's International Metabolic) database.

Gender equity improving among award winners and leaders at the Society for Investigative Dermatology.

Elevated Endogenous SDHA Drives Pathological Metabolism in Highly Metastatic Uveal Melanoma.

Metastatic uveal melanoma (UM) has a very poor prognosis and no effective therapy. Despite remarkable advances in treatment of cutaneous melanoma, UM remains recalcitrant to chemotherapy, small-molecule kinase inhibitors, and immune-based therapy.

and Activity of a Novel Antisense Peptide Nucleic Acid Compound Against Multidrug-Resistant .

Multidrug-resistant (MDR) is a difficult pathogen due to its propensity to develop resistance to antibiotics. Antisense nucleic acid analogs have been proposed as a potential alternative anti-infective approach. We developed a peptide nucleic acid (PNA) compound that targets the essential gene . The PNA oligomer was conjugated to the cell-penetrating peptide (RXR)XB. testing of the PNA conjugate against four clinical strains of MDR- in minimal medium demonstrated that all four strains were inhibited at a...

Public health-oriented intellectual property and trade policies in Africa and the regional mechanism under Trade-Related Aspects of Intellectual Property Rights amendment.

The short communication is prompted by the debate relating to the effect of pharmaceutical patents on access to affordable medicines, particularly in Africa. A recent amendment made to the Trade-Related Aspects of Intellectual Property Rights (TRIPS) Agreement creates a policy space for the regional alliance of low-income countries for the collective procurement and local production of drugs under compulsory licensing. This article examines the extent to which the regional mechanism can deliver access to ph...

Targeted mitochondrial delivery of antisense RNA-containing nanoparticles by a MITO-Porter for safe and efficient mitochondrial gene silencing.

Mitochondrial gene therapy will be needed to treat mitochondrial diseases. We previously demonstrated mitochondrial gene silencing by the mitochondrial delivery of antisense RNA oligonucleotide (ASO) targeting mtDNA-encoded mRNA using a MITO-Porter, a liposomal nano carrier system designed for mitochondrial delivery. Here, we report on the efficient packaging of ASO in the MITO-Porter via a nanoparticle packaging method, which showed a 10-fold higher packaging efficiency than the conventional method. The co...

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