leapcure’s Post

Why does the presence of JAK2 mutations lead to myeloproliferative neoplasms (MPNs) in some individuals but not in others?   A recently published study involving the collaborative expertise of researchers from @WellcomeSangerInstitute, @theUniversityofCambridge, and co-senior author Dr. @JyotiNangalia who serves on MPN Research Foundation’s Scientific Advisory Board, offers groundbreaking insights into the genetic intricacies of MPN development.  This pivotal research unveils:  The influence of inherited DNA on MPN susceptibility and disease progression.  A novel framework integrating genetic background with clinical manifestations.  “Previously MPNRF has funded our work exploring when in life the mutations that cause MPN are acquired. This study now sheds light on why such mutations result in overgrowth of bone marrow cells in some individuals more than others,” says Dr. Nangalia.  “I hope that in the future this information will help us predict which individuals are most likely to develop MPN if they are found to have a JAK2 mutation, and the type of MPN they may be predisposed to,” Dr. Nangalia adds.   Our mission is to fund innovative research that brings deeper understanding of these rare diseases to the MPN community. Bridging the gap between the unmet needs of those living with MPNs and existing preclinical research helps drive the discovery of effective and life-altering therapeutic solutions.   Dive deeper into these findings and join the discussion on how this shapes our understanding and treatment of MPNs.   [ Study abstract: https://lnkd.in/guXQuhzm ]  [ More on this study here: Understanding who develops which MPN and why – MPN Research Foundation ]  Join our community for more information on the latest research:

Precision medicine is revolutionizing RA care, offering personalized treatment plans based on your unique genetic makeup, environment, and lifestyle. Tools like PrismRA and Vectra are transforming the RA treatment landscape, helping patients find the most effective medications faster than ever before. With precision medicine, we can reduce the guesswork and tailor treatments specifically to your needs. But here's the catch: not all healthcare providers are familiar with precision medicine yet. It's up to us to advocate for ourselves and start the conversation with our doctors about these groundbreaking advancements in RA care. #PrecisionMedicine #RheumatoidArthritis #HealthcareInnovation #Advocacy

Published Today: From Advocacy to Early Cancer Detection and An Invited Presentation Before the 47th World Seminar on Women, Health, and Nursing For colleagues and friends, When we deal with issues surrounding chronic pain, may people have trouble focusing on anything else. Thus this reminder on Kevin MD, the most widely read healthcare newsletter in America: https://lnkd.in/gvjwB3nB From advocacy to early cancer detection I advocate for pain medicine regulation and share health care information on social media while emphasizing the importance of routine check-ups and early cancer detection based on my personal experiences... ================= Likewise, an invited presentation in today's plenary session of the 47th World Seminar on Women, Health, and Nursing: “Oversight on Revision of CDC Opioid Guidelines — A Process Predestined to Fail" Abstract: https://lnkd.in/g8GM_pKZ And the presentation as just posted: https://lnkd.in/gTDzeQdR I also wish to express my gratitude to Dr Jay Joshi who recorded the session for the Seminar and will also post it on his media platform, "Daily Remedy"

Despite comprising over half the world's population, women made up only 34% of Phase 1 clinical trial participants for FDA-approved drugs between 2013-2015. In a recent Clinical Leader article, Juan Camilo Arjona Ferreira, Organon's Chief Medical Officer & Head of R&D, highlights the enduring gender biases in drug development and explores opportunities to address current data gaps. The limited involvement of women in clinical trials contributes to expanding knowledge disparities, ultimately affecting health outcomes. The road to health equity is long, but the momentum is building. Join us and others in #womenshealth to make 2024 the year of transformation. 🚀 #WomensHealth #HealthEquity #HereforHerHealth #healthequity https://lnkd.in/enJhv7Us

#clinicalresearch #patient advocates #healthforall #skilling #awareness CanKids KidsCan Poonam Bagai Myelomafriends Aala Ansari Mohd Danish Khan Dr. Neha Singhal Dr. Ashish Bondia Nirbhay Singh Dr Sanjeev Singh Dr.Seema Pai Rhea Aggarwal Apar Health Durgesh Nandan Jha Dr. YK Gupta "Clinical research presents a promising path for improving healthcare in contemporary India. Yet, researchers identify gaps in trust, awareness, as well as misconceptions about being a ‘“guinea pig.” We proposed building the capacity of training patient advocacy groups (PAGs) in patient-centered clinical research and through them creating aware patients as research partners." Read more about the PACER (Patient Advocates for Clinical Research) efforts in our recently published paper https://lnkd.in/gp2vzSy2

Happy National Patient Advocacy Day! Today is an important day to recognize the important work done by patient advocates and their unique role in the transformation of individual and systemic issues in patient care. If you are unfamiliar with the type of work of patient advocates do, this is an excellent example. In this article from Forbes, CEO Rohit Nambisan outlines measures that researchers can take to make clinical research trials more patient-friendly and representative. By addressing barriers to care, research bias, and adopting a patient-centric approach in clinical research, the advocacy Rohit demonstrates in this article aligns with the goals of patient advocacy: to protect and promote the interests of patient populations. While patient advocates’ work can be on an individual, group, or system-wide scale, their efforts both protect the interests of their patients and increase healthcare equity. For example, by improving representation during clinical trials, or by promoting research and awareness for a rarely studied group of diseases. Their work is essential because an improvement in the inclusiveness of the healthcare industry at any level benefits the system as a whole. The more data we have and the more often we serve wider patient populations, the better care we can provide to anyone who walks through hospital doors. I have immense respect for patient advocates and their mission. I urge everyone who is interested in their work to get involved and support them. The future of healthcare and health policy is defined by our actions today. Do you agree? #healthcare #NationalPatientAdvocacyDay #HealthPolicy

Patients with rare diseases often take the initiative to navigate the healthcare landscape in order to access appropriate treatment for their conditions. Conducting clinical trials in the rare disease therapeutic area necessitates a shift from traditional recruitment methods, particularly due to challenges such as the absence of formal diagnoses and limited treatment options for patients. Empowering patients to engage in clinical trial programs for rare diseases can be significantly enhanced through the involvement of patient advocacy groups. This article from the Worldwide Clinical Trials team offers valuable insights into the obstacles faced by these patients and clinical trials sites, and proposes effective strategies for overcoming them. #clinicaltrials #raredisease #patienttreatment #medicalresearch #scientificinnovation #adaptivetrials #trialmethodology #Clinicalresearchorganisations

A Roadmap to Better Rare Disease Clinical Trials: 3 Tips for Effective Engagement with Patient Advocacy Groups https://lnkd.in/eAWX7g28 In rare disease research, organizations like Indo US Organization for Rare Diseases (IndoUSrare) are exploring effective strategies such as establishing clinical research sites in countries with a higher incidence of specific rare diseases. This approach, coupled with the utilization of AI-enhanced tools for clinical trial management and incorporating non-profit organizations as facilitators, provides alternative pathways for engaging patient groups. Although patient advocacy groups have traditionally been involved to improve patient recruitment and retention with mixed results, these additional strategies can be crucial for advancing research efficacy in the field. #raredisease #clinicaltrials #LMICs #India #globalcollaboration #AI #CTMS #clinicalresearch #clinicaldevelopment #patientregistry #patientadvocacy

Our Position Paper and Call to Action on anti-amyloid therapies for #Alzheimer’s disease has just been published as an Open Access article in the JPAD journal. In the Position Paper, which was developed in consultation with our member organisations and the European Working Group of People with #Dementia, we identify three priority areas where more work is needed: 📍 effective communication of risks and benefits 📍 an accurate, timely diagnosis; and 📍 healthcare systems preparedness. We also call for concrete actions to enable safe, timely and equitable access to these innovative medicines, for all people with early AD who could benefit from treatment, as well as continued investment in support, treatments and care services that can help people live well with #dementia at all stages. Many thanks to our colleagues at France Alzheimer for their contributions to the Position Paper, for translating it into french and making it accessible for broader audiences! Read the French translation, here: https://lnkd.in/g_sjbheB

🌍 Interview with HAE Junior on Cross-Border Patient Participation in Clinical Trials Our Co-Chair, Dr. Ingrid Klingmann, recently spoke with HAE Junior, discussing cross-border clinical trial participation for European patients.  In this interview, Dr. Klingmann highlights the experiences of patients involved in cross-border trials and explains how the EU-X-CT initiative aim to improve the current status quo. Hereditary Angioedema (HAE) is a rare genetic disorder that causes severe and unpredictable swelling. HAE Junior supports affected patients and their caregivers. 📖 Read the full interview: - English version: https://lnkd.in/e8u94pwh - Czech version: https://lnkd.in/eMK2ac6b A heartfelt thank you to HAE Junior for their continued collaboration and dedication to patient empowerment. 💙 #ClinicalTrials #PatientEngagement #EUHealth #CrossBorderHealthcare #HAEJunior #RareDiseases