Alltrna’s Post

🔑✨ TOP PSORIASIS TAKEAWAYS FROM AUGUST: #PsoriasisAwarenessMonth was packed with updates on psoriatic disease: Alumis Inc.’s ESK-001 phase 3 trial, the impact of green spaces on risk, and apremilast becoming available for pediatric patients. Explore our comprehensive recap to stay informed on these important developments and more. ⬇️ https://lnkd.in/gtmadrk9

In the 📕#RedJournal: Adalimumab vs Infliximab in Luminal Pediatric Crohn's Disease: Comparable Outcomes in a Prospective Multicenter Cohort Study deBruyn, et al. 👉 bit.ly/3vCbmD5

Our latest Whitepaper, 'Navigating the Rare Pediatric Disease Priority Review Voucher Program, Ahead of September 2024', is now available to read and download, https://lnkd.in/g8SdRH9E - The paper, written by Iheoma Anosike, provides useful information relating to The rare pediatric disease designation and RPD PRV program, with important considerations for Sponsors. For support ahead of the upcoming 30 September 2024 sunset date, get in touch to speak with a member of the team www.scendea.com/contact - #whitepaper #drugdevelopment #regulatoryaffairs #pediatricdisease

As a voice for the rare disease community, a recent op-ed in STAT really hit home for me. The article focuses on how the FDA’s rare pediatric disease priority review voucher program will disappear unless Congress reauthorizes it before September 30. As NORD's Pamela Gavin shares in the byline, more than 30 million Americans live with a rare disease, and nearly half are children – the program disappearing would be a devastating end for patients and their families eagerly waiting for treatment.    At Aldevron, we are committed to advocating for individuals with #rarediseases by helping develop the tools to create breakthrough treatments, and we understand how important increased funding and programming is in this often-forgotten space. We must continue to push the government to incentivize #drugdevelopment for rare diseases, ensuring that patients continue to have hope for their future. #PRVs #RareDisease #FDA 

Today at 2 pm ET. Learn how you can help preserve this important program to incentivize research on rare pediatric diseases.

In this short video, Dr. Leslie Gordon, Co-Founder and Medical Director of The Progeria Research Foundation, explains the vital importance of Rare Pediatric Disease PRVs for #progeria and other #RareDiseases. If you have not taken action on this issue, do so here: https://lnkd.in/ettvJA2p

U.S. President Biden signed a bill postponing the sunset date of the rare pediatric disease Priority Review Voucher (PRV) program from September 30, 2024, to December 20, 2024. The bill, the “Continuing Appropriations and Extensions Act, 2025” is a temporary measure to continue the rare pediatric disease PRV program. There are currently other ongoing legislative efforts to renew the program through 2028 and later. We have summarized these proposals here: https://lnkd.in/exn-tcha #PediatricHealth #PriorityReviewVoucher #ContinuingAppropriationsandExtensionsAct2025 Authors: Komal Karnik Nigam, Deborah Cho, and Bryan Walsh.

Earlier this week, the Global Commission to End the Diagnostic Odyssey for Children with Rare Disease re-launched with a clear plan for improving and accelerating diagnosis, a process that can be painfully long —5+ years for some children.   Sanofi is proud to serve as co-chair of the Global Commission alongside EURORDIS-Rare Diseases Europe and Takeda. Together, the Global Commission envisions a world where a clear path to a timely, accurate diagnosis exists for all children living with a rare disease.   The Global Commission is developing a framework and resource compendium of case studies that have been shown to successfully reduce the diagnostic odyssey. By publishing and sharing these stories of success, we hope to improve access to timely and accurate diagnoses for children around the world. Read the first iteration of the 2024 Framework for Action report: https://lnkd.in/dXF2N9tA   #raredisease

Today, on Rare Disease Day, we at ConSynance Therapeutics stand in solidarity with the rare disease community. Rare diseases affect over 300 million people worldwide, yet they often receive less attention and research funding compared to more common conditions. This day is a reminder of the importance of inclusivity in healthcare and the need for continued research and development in the field of rare diseases. At ConSynance Therapeutics, our team is dedicated to the discovery and development of innovative treatments that can change the lives of those affected by rare diseases. We recognize the resilience of patients, caregivers, and healthcare professionals who navigate the complexities of rare diseases daily. Your strength inspires us to persevere in our quest for solutions that can make a significant difference. We also want to extend a heartfelt thank you to the patients participating in clinical trials. Your courage and commitment are crucial for developing new treatments. Your participation provides hope for not only your future, but the entire rare disease community. Let's use Rare Disease Day to amplify the voices of the rare disease community. Together, we can make a difference. #rarediseaseday2024 #rarediseaseday #ConSynanceTherapeutics #rarediseases #clinicaltrials #thankyoupatients

Do you know that all pediatric oncological diseases are considered rare diseases? Yes, this is right, and if it wasn't for the #fda and #ema requirements, maybe most of these diseases wouldn't be ever researched on children and adolescents. Today as we acknowledge #rarediseaseday2024, let's thank all people fighting for better diagnosing and treatment access to these patients and their families. Let's pray for all people going through this journey to find their treatment. Let's hope that one day no matter how rare the disease is, we will know how to fight it. I will use the opportunity to mention that if you are going through a rare disease today or your close one is, at FindMeCure we can help you free of charge to search among all available #clinicaltrials and information about available treatments and specialists. Ping me or the team if you need help! #OrphanDrugs #PersonalizedMedicine #ClinicalTrials #NGS #HealthcareInnovation #PatientAdvocacy #DrugDevelopment #TrialHub #FightRareDiseases #FindMeCure