Prader-Willi Syndrome Association | USA’s Post

CAR-T therapy in CLL could be an area where biomarkers would be helpful.  The FDA recently approved lisocabtagene maraleucel in CLL/SLL after at least two prior lines of therapy, including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL2) inhibitor.  Looking at the published data of only the 49 patients who received lisocabtagene maraleucel at the FDA-approved dose, 9 patients (18.4%) had a complete response (CR), and those patients, at median follow-up of 20.8 months, were all progression-free.  12 patients (24.5%) had a partial response (PR) or nodular partial response or remission (nPR), and those patients had median PFS of 26.2 months,   Lisocabtagene maraleucel also appears to have an increased risk of adverse events in CLL. For example, cytokine release syndrome (CRS) occurred in 83% (74/89) of all patients, including grade 3 CRS in 9% of patients, much higher than in the DLBCL patients.  In the BRUIN trial, pirtobrutinib had a median PFS of 16.8 months in the BTK/BCL2 refractory population.  However, pirtobrutinib had zero patients with a CR.   Given the cost and increased adverse events of lisocabtagene maraleucel, it would be nice to have biomarkers that could identify which patients could achieve a CR.  If the CR data holds up and we are curing patients, we want to be able to do so - but it would be difficult to recommend this to all patients to whom this indication would apply to. #breyanzi #jaypirca #cll https://lnkd.in/eaii6qvv

Zynerba Pharmaceuticals’ RECONNECT trial now has a completely at-home/virtual option for participation in addition to the original option to participate at clinic site! Several families have already enrolled in the RECONNECT trial using the at-home/virtual option and have appreciated the flexibility and convenience. The RECONNECT at-home trial option has eliminated barriers for families who previously could not participate due to travel or behavioral challenges. Families who have had significant challenges going to a clinic for visits have also enrolled. Learn more here:https://hubs.ly/Q02gbYlL0

🌟 May 16th is hae day :-) Hereditary Angioedema 🌟 📅 Today, let's reflect on the incredible strides we've made in advocating for better therapies and an improved quality of life for those affected by Hereditary Angioedema. 🚀 I'm thrilled to share that we are currently witnessing an unprecedented number of effective therapies, with eight FDA-approved products already available and an impressive thirteen more in development. This progress is a testament to the dedication and hard work of countless researchers, clinical teams, and advocates in our community. As we continue to build momentum, let's harness the galvanizing force of HAE Day to fuel the ongoing quest for new and improved treatments. 🙏 A huge thank you to all the researchers and clinical teams who tirelessly dedicate themselves to advancing HAE treatment options. Your efforts are truly changing lives. Let's keep pushing forward! #haeday #HAEawareness #citeline Astria Therapeutics, Inc. Hereditary Angioedema Association - HAEA #clinicalresearch #clinicaltrials #clinicaldevelopment

Intended for HCPs with an interest in GEA:   At Jazz, we believe all people, including those who live with complex conditions with limited options, deserve new therapeutic options to help improve outcomes.   The HERIZON-GEA-01 clinical trial seeks to explore the safety profile of our investigational treatment for people living with advanced/metastatic human epidermal growth factor receptor 2 (#HER2) positive gastroesophageal adenocarcinomas (#GEAs) and learn if it’s more efficacious than the current standard of care.   This trial is currently enrolling eligible patients, learn more: https://lnkd.in/g8RNyyQ8

How can we help accelerate your oncology clinical trial milestones? Meet with my LabConnect colleagues during #ASCO to find out!

💡 Breaking Barriers in Rare Disease Research - From development planning to regulatory submissions, ERGOMED partners with you to design and execute innovative, patient-centric clinical research programs for patients and families living with the challenge of a rare disease. Ready to learn more? Meet our team at Booth #437 at World Orphan Drug Congress USA 2024 next week! Together, let's make a difference. #RareDisease #PatientCentricity #Innovation #ClinicalResearch

Proud to announce that Mermaid Bio GmbH and their CEO Ranja Salvamoser will be presenting at Meet the DONs Virtual Event on the 24th of October. Sophisticated investors, decision makers, and opportunities are all brought together by this special online experience. Due in part to COVID, we have welcomed over 1,500 virtual attendees since our launch in 2020. A true meeting place for CEO's, Founders, decision makers and sophisticated investors. Mermaid Bio GmbH’s Intrabodies represent a novel precision-medicine approach that can tackle notoriously difficult targets and is piloted directly to the tissue of interest to avoid damaging side effects. Intrabodies The power of antibodies and targeted drug delivery, wrapped in one. By pairing antibodies with intracellular delivery, Intrabodies bring the strength of antibodies into diseased cells, without damaging healthy tissues. Beyond giving patients new hope, our purpose is to restore their quality of life If you are a Founder, CEO or sophisticated investor register for free with this link - https://lnkd.in/eRMU3CEH Are you a healthcare business seeking funding if so, feel free to submit your opportunity to our network via this link - https://lnkd.in/ep8Bqsb8

How can we help accelerate your oncology clinical trial milestones? Meet with my LabConnect colleagues during #ASCO to find out!   

This month to celebrate the "Love Drug", we're running a 30% off sale our on our in-depth online course "Foundations in MDMA Safety, Therapeutic Applications & Research". If exploring MDMA is of interest to you, now is the time to jump into the course! If you'd like to explore some topics we cover first, here are the top 10 articles on our site about MDMA: https://lnkd.in/gsvFHZ-q

Today I’m so proud to share the full version of Alexion Pharmaceuticals, Inc.’s first short film, Rare Connections in NMOSD, which centers the voices of our patients and their families. I hope you will join me in making an impact in the months to come by sharing this film and these stories, to foster connections and spark important conversations. In Rare Connections we hear from Marie, Alex, and Craig, who share the strength, resilience, and beauty of their community, along with the significant challenges they experience living with NMOSD. While every rare disease patient has their own unique story, many of them share a similar, and often difficult, journey. From a lengthy quest to get a proper diagnosis to difficulty accessing quality care, these are the all-too-common stories that underscore the need for policy changes and better care access opportunities for patients. We can only achieve this through close collaboration with the entire rare disease community. This is why at Alexion Pharmaceuticals, Inc. we are focused on improving equity and health outcomes for people living with rare diseases by working to reduce the time to diagnosis, improve access to care, and engaging with global policymakers to amplify impact. Many thanks to Elizabeth Preston, Julie Block, the Alexion Patient Experience team and everyone across our organization who worked so hard to make this film a reality. https://lnkd.in/d5KRd9ip