Verona Pharma’s Post

Discover the challenges and opportunities in the rare disease market with our latest white paper. Gain insights on launching orphan medicines, overcoming hurdles and achieving #launchsuccess. Download now to learn from successful #RareDisease launches. https://bit.ly/3yaeo2u #LaunchExcellence

ICT Spring 2024 is now live! ☀️ Featuring insights in Decentralised Clinical Trials, Orphan Drug and Rare Disease Trials, and much more! Read here: https://bit.ly/3URlpxX #ClinicalTrials #decentralisedclinicaltrials #rarediseases #orphandrugs #pharmaceuticalindustry

Following 2023’s successful EL-PFDD meeting, our new Voice of the Patient Report is a critical next step in the IPPF’s engagement with FDA and other decision-makers. As we’ve learned, there are significant obstacles facing #pemphigus and #pemphigoid patients’ access to potential future therapies. To overcome these obstacles, we must empower the voice of a strong coalition of patients, caregivers, researchers, and clinicians so that regulatory agencies and industry partners understand what truly matters to people living with these diseases.

Getting a lot of positive reaction with our new white paper on Rare disease Launch Excellence..see below.

"Making the most of real-world data from disease registries across the product lifecycle" As most were winding down for the holidays and might have missed it, I'm resharing this fantastic blog by Tsitsi Monera-Penduka, PhD. 🎉Happy New Year all!🎉 #realworldevidence #registries #rwd

Gain insights on launching orphan medicines, overcoming hurdles and achieving #launchsuccess. Download now to learn from successful #RareDisease launches. https://bit.ly/3yaeo2u

ICT Spring 2024 is now live! ☀️ Featuring insights in Decentralised Clinical Trials, Orphan Drug and Rare Disease Trials, and much more! Read here: https://bit.ly/3URlpxX #ClinicalTrials #decentralisedclinicaltrials #rarediseases #orphandrugs #pharmaceuticalindustry

Did you know that today is #RareDiseaseDay? Check out Certara Talks with our experts, Oxana Iliach, Rajesh Krishna, David Wesche, and Paul Martin, who discuss the combined efforts and expertise needed for a successful rare disease drug submission. They also share insights into the regulatory challenges that rare disease development programs need to think about. ▶️ Watch now: https://ow.ly/CkGc50QH9HZ Curious to delve further into the world of rare disease drug development? Explore more at: https://lnkd.in/eTDeSvhH #RareDiseaseDay #DrugDevelopment #CertaraTalks

Plan to be in #CPHINorthAmerica2024 #CentralAsia #CPHINorthAmerica2024 #orphandrugs #PharmAccess

Weekly Rare Update People living with a rare disease in the UK face daunting hurdles: lengthy delays to diagnosis, few or no treatment options, and inequitable access to care they need. Whilst measures are being taken to improve health equity for this community, there is more to be done. Last week, on Rare Disease Day 2024, I've contributed to the Guardian Rare Disease supplement discussing on how AI offers hope to streamline diagnosis including the work undertaken with Mendelian, and how policy reforms and collaborative efforts are crucial to improve the pathway for patients with rare diseases. I firmly believe that through advances in technology, and through multidisciplinary collaboration, we can find solutions and effect positive change for the people living with rare diseases.   #RareDisease #RareDiseaseDay Alexion Pharmaceuticals, Inc.