Rhythm Pharmaceuticals Inc.’s Post

When it comes to immune-mediated diseases, Maria Belvisi, Senior Vice President and Head of Research and Early Development, feels it is imperative that we find ways to "intervene earlier and stop progressive tissue damage" as we focus on uncovering new approaches and advancing remission. Discover more: https://learn.az/60495zUUd #EULAR2024

There is an urgent need to transform care and improve outcomes in immune-mediated diseases, like lupus and RA, as patients struggle to achieve disease control. At AstraZeneca, we have a bold ambition to make remission a goal. Learn more about how we’re advancing the science to make that a reality.   #EULAR2024

Today, on International Rare Disease Day, we stand together to raise awareness and ignite change for the 300 million people worldwide who live with rare diseases. Rare diseases pose unique challenges and are often caused by genetic factors, affecting both children and adults. Tragically, more than 30% of children with a rare disease do not survive beyond their fifth birthday. These patients and their families face a lifelong struggle, as these conditions are rarely curable. At IQVIA, we recognize the urgency of addressing rare diseases. Our commitment extends beyond scientific curiosity, it’s about making a real difference in the lives of those affected. Learn more about the work we do at IQVIA in the fight against #rarediseases: https://bit.ly/3TfURpj

This #RareDiseaseDay join us as we raise awareness about the importance of having a collaborative approach among governments, healthcare stakeholders, and #patient support groups to increase awareness about rare diseases, improve patient identification and diagnosis, and provide access to affordable treatments. Learn more about the work we do at IQVIA in the fight against #rarediseases: https://bit.ly/3Ik2i8W

Scientists are actively working to single out therapies that have potentially better health outcomes for people living with inflammation from atherosclerosis and chronic kidney disease.   This necessary research today could mean a brighter future for our community on the horizon.   Our community’s voice matters in these important decisions and discoveries. Take part in making a brighter future by ensuring adequate representation in clinical trials.   Learn about how African Americans benefit from participating in clinical trials at https://bit.ly/3RrW1xt. #blackinclinicaltrials #blackhealthmatters

This snapshot of the content of our recent paper helps highlight the unmet need of those living with FCS. The devastating impacts of acute pancreatitis events and the broader long term life impacts. Currently the only treatment involves dietary restrictions which add to the negative impact on quality of life. Hopefully recent positive phase 3 trial results will translate into more options for the FCS community.

Over 10,000 rare diseases impact over 30 million Americans and their families. Given the uncommon and uncertain nature of rare diseases, individuals with rare diseases face significant challenges in diagnosis and treatment.  Limited diversity and inclusion in rare disease research hinders opportunities for care and contributes to poorer outcomes.  Gain our expert insights on the case for the significance of DEI rare disease clinical trials and participants in our DE&I section of the highly anticipated Clinical Trial Trends & Insights 2024 from WCG! Download now at https://lnkd.in/eB8ptAWe #DEI #2024 #clinicalresearch #clinicalresearchtrends #raredisease #WCG 

Although rare disease affects a small number of people within a population, the impact is large. According to the National Center for Advancing Translational Sciences, there are over 10,000 known rare diseases. As rare disease relies on science and patient perspective, the day-to-day journey can be eased with improvements on guided plans, and funding programs that enhance education, real-time patient resources and wellness programs for the rare disease community. #raredisease #rarediseases #patientresources #telehealth #patientnavigation #healthinnovation

"Taken together, rare diseases affect an estimated 300 million people globally so the experience of having a rare disease is, sadly, not particularly rare.” A disease is considered rare when it affects fewer than one in 2000 people. The associated challenges with this are many, including delays in diagnosis, challenges in accessing appropriate care, high personal financial cost of treatments and fragmented research to name a few. Rare Disease Day is an international initiative with an objective to work towards equity in social opportunity, health care, and access to diagnosis and therapies for people living with a rare disease. https://lnkd.in/egkyMSnx

🌟 Today, on Rare Disease Day, PharmaKinnex proudly stands shoulder-to-shoulder with our clients, embarking on a shared journey to confront the unique challenges posed by rare diseases. We recognize the profound importance of offering steadfast support and pioneering solutions to individuals navigating the intricate landscapes of rare conditions. 🔍 At PharmaKinnex, we delve deep into the complexities of rare diseases, armed with a deep understanding of their nuances and the pressing need for innovative interventions. Our commitment knows no bounds as we strive to provide tailored support and groundbreaking strategies that empower our clients to navigate these uncharted waters with confidence. 💪 Today, and every day, we draw inspiration from the extraordinary resilience and unwavering courage displayed by those confronting rare diseases. Their indomitable spirit fuels our resolve, propelling us forward in our mission to stand by their side and champion their pursuit of better health and brighter futures. 🤝 Together, let's amplify our efforts to raise awareness, drive progress, and effectuate tangible change in the lives of those affected by rare diseases. By fostering collaboration, igniting advocacy, and embracing innovation, we can forge a path towards a future where rare diseases are met with understanding, compassion, and effective solutions. #RareDiseaseDay #SupportingOurClients #MakingADifference