Amgen Inc. is sponsoring a global, phase 3, double-blind cardiovascular outcomes study titled the OCEAN(a)-Outcomes Trial (The OCEAN(a) (Olpasiran Trials of Cardiovascular Events And LipoproteiN(a) Reduction – Outcomes Trial) to compare the effect of treatment with with investigational drug olpasiran to placebo, on the risk for coronary heart disease death (CHD death), myocardial infarction, or urgent coronary revascularization in participants with atherosclerotic cardiovascular disease (ASCVD) and elevated lipoprotein(a).
Participants will be randomly assigned to receive olpasiran or placebo administered by subcutaneous injection once every 12 weeks at the study site. The study drug will not replace any existing treatments that the patient may be receiving. The average follow-up duration will be around 4 years. The study is currently recruiting participants.
Key study requirements:
Adult patients receiving stable, optimized lipid-lowering therapy must have a history of myocardial infarction and/or percutaneous coronary intervention and an Lp(a) value of ≥ 200 nmol/L at screening by central laboratory.
From the abstract: " What genetic modifiers of sickle cell disease (SCD) are currently defined, and what are potential approaches to improve future studies? In this systematic review and meta-analysis of 571 studies examining 29?670 individuals with SCD, 17?757 associations involving 1552 genes and 25 SCD phenotype categories were discovered; of these, only 173 associations met the study design, reporting, and phenotype or genotype harmonization required for meta-analysis. Gene variants regulating fetal hemoglobin and a-thalassemia were frequently identified, but other associations remained unconfirmed."
NHLBI is refreshing its Strategic Vision to address emerging scientific opportunities in accordance with new NIH strategic plan requirements. Help identify scientific priorities, make decisions, and allocate resources by sharing your input.
As part of our work within the HTx Project, EURORDIS has developed the HTx Patient Toolkit - a series of resources and educational videos developed for advocates on health technology assessments (HTAs) and research conducted through the HTx project. François Houÿez, our Information & Access to Therapies Director, is among the instructors featured in the video series.
Along with our Together4RD project partners, Matt Bolz-Johnson, our Mental Health Lead and Healthcare Advisor, and Ines Hernando, our ERN and Healthcare Director, are among the co-authors who recently published a position statement calling for greater collaboration between European Reference Networks and industry.
Should more diseases be screened for at birth?
In our latest episode of Rare on Air, Julien Poulain talks to Iuliana Dumitriu, the mother of Victor, an eight-year-old boy living with Coffin-Lowry Syndrome in Romania. Iuliana, who is also the President of the Coffin-Lowry Syndrome Association shares with Julien her family’s story of the torturous, yet determined, seven-year journey toward receiving a diagnosis for Victor. Julien also explores the results of our recent Rare Barometer survey with our Social Research Director Jessie Dubief.
Gene Age Exhibition
This month in Prague, the Association of Gene Therapy launched its Gene Age exhibition, accessible both in-person and online. The exhibition is aiming to heighten awareness and education regarding the impact of genes and their mutations in the development of human diseases, with a special focus on rare diseases. EURORDIS is a partner of the exhibition.
Have you completed the EURORDIS Access Campaign Questionnaire? We are inviting anybody living with a rare disease to share the difficulties they may be facing in accessing medicines, medical devices, surgeries, or medical consultations. The responses will help us uncover the hurdles to treatment that exist, toward the aim of improving access to treatments across Europe. Complete the questionnaire by 30 October.
The Social Policy Action Group (SPAG) will support EURORDIS’ work to shape policies and practices that improve the access of people living with a rare disease and their families to holistic care, and the guarantee of their social and human rights.
In Barcelona last week, Gulcin Gumus, our Senior Manager for Research & Policy, led a meeting of the Screen4Care Forum on Newborn Screening. With more than 40 rare disease experts and project partners in attendance, the meeting delved into various topics, including the challenges of newborn screening research versus implementing practices of screening at birth into national health systems.
On 28 November at the European Parliament in Brussels, EURORDIS and Mental Health Europe, as well as MEPs Sara Cerdas and Tilly Metz, will be hosting an event on the European Commission's recent Comprehensive Approach to Mental Health. The event will address how and why the Commission's Approach must support the mental health of individuals, including those with rare diseases, who face distinct challenges to their psychological wellbeing.
The ECRD is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place.
Leading, inspiring and engaging all stakeholders to take action, the Conference is an unrivalled opportunity to network and exchange invaluable knowledge with over 1500 stakeholders in the rare disease community – patient advocates, policy makers, researchers, clinicians, healthcare professionals, healthcare industry representatives, academics, payers, regulators and Member State representatives.
The next ECRD will take place as a fully hybrid conference on 15-16 May 2024, in Brussels and online!
Women, power, and cancer: a Lancet Commission
Nov 13, 2023
14:00 GMT / 9:00 EST
Women, power, and cancer: a Lancet Commission was created to address urgent questions at the intersection of social inequality, cancer risk and outcomes, and the status of women in society. In this Lancet Webinar, authors of The Lancet Commission will summarise the key findings of the Commission, including cancer equity and gender transformative policies, cancer prevention in women, gender-responsive frameworks in the healthcare context, and the value of women in cancer economics.
Directing vaccines to dendritic cells
Many cancer vaccines under investigation use mRNA to deliver key information to the immune system. But most of these trials do not effectively target dendritic cells, a key orchestrator of the immune response. Because dendritic cells display antigens on their surface, which activates other components of the immune system and sounds the alarm about a potential threat, they are an attractive target for cancer treatment strategies.
With the ultimate goal of developing an improved vaccine strategy, a research team at Johns Hopkins University has designed a targeted, biodegradable nanoparticle that specifically delivers mRNA to dendritic cells. When evaluated in multiple mouse tumor models, treatment with the nanoparticles provided robust antitumor activity when combined with a type of therapy called immune checkpoint blockade. Their results were recently reported in the journal PNAS.
ver historia personal en: www.cerasale.com.ar [dado de baja por la Cancillería Argentina por temas políticos, propio de la censura que rige en nuestro medio]//
weblog.maimonides.edu/farmacia/archives/UM_Informe_Autoevaluacion_FyB.pdf - //
weblog.maimonides.edu/farmacia/archives/0216_Admin_FarmEcon.pdf - //
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