Citryll’s Post

Are you in the drug development business and in Vienna this week? Come meet Elad Berkman and Dan Goldstaub! They will tell you how ML and adaptive design are combined to accelerate Rheumatology trials

Discover the Power of Interdisciplinary Care with seca mBCA North America and inHealth Lifestyle Therapeutics! This webinar on March 29th will show you how to fill the void in traditional obesity care through our collaborative efforts. Get insights from the SECA and inHealth Lifestyle Therapeutics experts!

Check out the latest Our Stories as I had the honor to write about our Senior Vice President, Global Head of Precision Medicine, Dale Shuster. It's been so interesting getting to know him.

6th edition already… a must-attend, human-sized conference on Digital health in Rheumatology where interactions and expertise are at the center of the event.

In the world of Duchenne research, collaboration and patient-centered approaches are crucial. Parent Project Muscular Dystrophy has embraced Clinical Trials Transformation Initiative (CTTI)’s Chevron Diagram to deepen the integration of patient voices into every phase of drug development. This approach has already led to significant progress, with seven patient preference studies completed, providing invaluable insights that are shaping the future of Duchenne disease. Learn more about PPMD’s successful collaboration with CTTI at https://bit.ly/PPMDcs.  

Today is Rare Disease Day at The National Institutes of Health! Stream the conference live here https://buff.ly/49MRWtI. The agenda is filled speakers from Congress, the FDA, Industry, Academia, and patient advocates all coming together to discussing ways to accelerate development of new therapeutics for this underserved population. We are proud to have several members of our team presenting at the event, specifically: James J. Hickman, Ph.D. will be speaking on the main stage at noon ET on how Microphysiological Systems (MPS) are currently supporting rare disease therapeutic development. Christopher Long, Kaveena Autar, and Xiufang Guo will be presenting posters on the following topics, respectively: "Implementation of a Human Cell-Based Malaria-on-a-Chip Phenotypic Disease Model for Drug Efficacy Evaluation" "Evaluation of Long-Term Potentiation and Drug Efficacy in a Familial Alzheimer’s Disease hiPSC-Cortical Neuron Microphysiological System" "A human-based functional NMJ system for personalized rare disease modeling and drug testing" #NIH #RareDiseaseDay #MPS #RDDNIH #OrganOnAChip #HumanOnAChip

Great to hear about a really positive story coming out from a type of funding under the #EJPRD (European Joint Programme for #RareDiseases ) This afternoon Bert Smeets presented very nicely on the multistakeholder collaborations set in motion for #LAMA2 Muscular Dystrophy, enabled by the 'EJPRD Networking Scheme'. Joanne Lee and I have also managed to secure funding through this valuable scheme in the past, which we used to advance our efforts to spread a powerful model for better clinical trials into more rare diseases. The model was created in the #Neuromuscular community under TREAT-NMD , many years ago, and our work in EJP-RD has been to try to expand its use outside of this area. We were able to complement our toolkit (https://shorturl.at/9HXbu) with a 2023 workshop aimed at ERN RARE-LIVER ERN RARE-LIVER and ERN GUARD-Heart in particular, to encourage them to think about how such a model could improve clinical research in their areas. Meanwhile, with support from Joanne Lee and colleagues in the John Walton Muscular Dystrophy Research Centre, there is now an ACT (Advisory Committee for Therapeutics) in the #ataxia field operated by the Ataxia Global Initiative (find out more here - https://lnkd.in/eDWwbMap and here https://shorturl.at/JYQ1k )! Great to see things which work in one rare community being transplanted into others :-) The Networking Scheme -https://shorturl.at/2iOyn- will continue under the European Rare Disease Partnership, #ERDERA so look out for opportunities to bring diverse stakeholders together to drive forwards RD research!

Vivli Researcher Spotlight: 🔦 Check out our newest update, with researcher Dr. Ricardo Ferreira on using Vivli to access #clinicaltrialdata, and proposing a more #patientcentered approach to managing #rheumatoidarthritis: https://lnkd.in/gGzxcJVc #RA #datasharing

The #EU Health Technology Assessment (#HTA) Regulation will apply from 12 January 2025 to #oncology products and #ATMPs, including #OrphanMedicinalProducts (OMPs) with these indications. From 13 January 2028, the new procedure will apply to all OMPs, irrespective of the therapeutic indication. 💻Join us for the fifth episode in our #LifeScienceLectures webinar series, where we’ll discuss challenges in evidence generation for #OMPs and experiences with national HTA procedures, to explore possible methodological approaches that will ensure joint clinical assessments (#JCAs) speed up #access to treatments for people living with #RareDiseases. 🔗 Register here: https://lnkd.in/dp_XMUgV Our panel: 🗣️ Maurizio Scarpa, Coordinator, ERN for Rare Hereditary Metabolic Diseases (MetabERN) 🗣️ Silke Honsek, Senior Manager, International Market Access & New Assets, AOP Health 🗣️ Mark Sheehan, Associate Professor, NIHR Oxford Biomedical Research Centre 🗣️ Sheela Upadhyaya, Life Sciences Consultant Specialist 🎤Moderated by EUCOPE's Alexander Natz 📅 3 June 2024 (16:00 - 17:00 CET) 📺 The new EU HTA procedure: How can JCAs support access to OMPs for people living with rare diseases

Join us for a free UCB-sponsored webinar on Wednesday, April 24, 2024, with Wendy Cantrell, DNP, CRNP, Kara Gooding, MMS, PA-C, Joe Gorelick, MSN, FNP-C, and Andrea Nguyen, PA-C, MPAS. - Get NP/PA perspectives and real-world insights as panelists present and discuss their own patient cases managed with IL-17 inhibitors. - Learn about the science of IL-17 inhibition and key therapeutic considerations for this growing class of biologic agents. - Explore the latest data on treatment outcomes with a focus on patient and prescriber expectations of “treatment success.” - Discover key considerations related to patient selection and screening, as well as treatment initiation and monitoring. Register for FREE: https://lnkd.in/eyckBZUu