Orchard Therapeutics - U.S.’s Post

With favorable safety and promising efficacy, we’re continuing dose-escalation in our CAHptain-205 study, investigating #tildacerfont for the treatment of classic Congenital Adrenal Hyperplasia (#CAH) in children 2+ years of age and adults. Learn more about CAHptain-205: https://lnkd.in/eRH2P23Y  

This March, during Multiple Sclerosis Education Awareness Month, Medvantx is raising awareness for the 1.8 million patients worldwide that are struggling with this disease. Multiple Sclerosis (MS) affects the central nervous systems, severing essential communication lines within the body, causing potential disabilities. We are grateful to have dispensed nearly 31,000 orders, last year, from our independent pharmacy, helping to increase patient access for four vital MS medications. Our patient assistance programs fulfill our mission, offering much-needed support to those encountering MS. We understand the ins and outs of this complex condition and strive to provide not just medications, but hope and a connection to therapy. Share this message to raise awareness! https://lnkd.in/gqJKvZuF #Medvantx #MSconnections #PatientAssistancePrograms #Pharmacy #DispensingMeds #AccessibilityForAll #HealthcareAccess

This week concludes Peripheral Neuropathy Awareness Week 2024. Peripheral neuropathy affects 190 million here and around the world, however, it is still not well known or understood. The Foundation for Peripheral Neuropathy is a support system that provides videos, brochures, and public service announcements, demonstrating that patients are not alone. Learn more 👉 https://hubs.la/Q02vzk_b0 #peripheralneuropathy #foundationforpn #peripheralneuropathyawarenessweek #SimplifyingComplexCare #SpecialtyPharmacy

🚨 New ACTION publication 🚨 Using data from the ACTION registry, authors reviewed outcomes for patients who received Impella pumps alongside VA-ECMO from 2014 to 2021. The results? Out of 20 patients, 45% recovered, 40% moved to durable VADs, and 5% got heart transplants. These findings suggest Impella support could be vital for older pediatric patients to help their hearts recover or prepare for the next steps in treatment. Learn more about their study here: https://bit.ly/3ysUpfD! #PedsHF #PedsVAD

The SHINE trial outcomes demonstrate that infants receiving high velocity therapy achieved a higher initial intubation success rate compared to those under Treatment as Usual (TAU). Delve into the study findings for further insights >https://hubs.ly/Q02qBxj-0

Less than two weeks after EASD we go again. We meet again, we talk again, we learn more. And what a special one it is, this year’s INTERNATIONAL SOCIETY FOR PEDIATRIC AND ADOLESCENT DIABETES (ISPAD). Not only because it is my first edition, but also it is close to home, in Rotterdam. But mostly as the congress focus is almost completely on #type1diabetes. It also brings together many parts of my life with #aT1D. The past: in JDRF Nederland, and now co-organizing a wonderful event on the need for screening for aT1D. The present: meeting up with a medical psychologist from Diabeter, where I’ll be participating in a study on nocturnal hypoglycaemia.  The future: learning about research outcomes that will change the way we view treatment of aT1D.    If this is 10 years of living with aT1D, it's not all bad - and it makes me hopeful for the next decade. 

🌍👁️ World Glaucoma Day: Spotlight on Sight with CR Integration 👁️🌍 Today, on World Glaucoma Day, let's uncover the unseen truths about this sight-threatening condition. Did you know that glaucoma affects more than 76 million people globally, with nearly half unaware of their condition? It's often symptomless until significant vision loss occurs. At CR Integration, we're committed to raising awareness and supporting vital clinical research initiatives. Early detection is key in the fight against glaucoma, and advancements in clinical research pave the way for better treatments and diagnostic tools. Join us in spreading awareness, promoting early detection, and supporting ongoing research efforts. Together, we can make a difference in preserving sight and improving lives. #WorldGlaucomaDay #CRIntegration #EyeHealth #Awareness #ClinicalResearch #EarlyDetection

Our Meta-Analysis is out... and Peds Integrated Primary Care Still Works! Our meta-analytic sample of 6,897 found: 1. Peds IPC is superior to enhanced usual care at improving behavior, quality of life, and symptoms. 2. Trials assessing treatment and prevention models were both effective 3. Co-located and Highly Integrated models were similarly effective We also found we have a lot of work to do in standardizing how we report our models and outcomes in Pediatric IPC. So honored to be part of this group: Jeffrey Shahidullah, Jennifer Mautone, PhD, Tiffany Rybak, Chimereodo Okoroji, PhD, NCSP, Teryn Bruni, Kevin Stephenson, Leah Vance Utset, @Kurt Freeman, @Leah Lalonde, Andrew Riley Check it out here: https://lnkd.in/g8ZkpaEk

There is a new kind of pioneer; this film focuses on two patients as they undergo the first year of a clinical trial; and showcases how scientists and patients work together to test a possible cure for diabetes. #clinicaltrials #healthresearch #healthresearchbc #innovativemedicine #diabetes

Our latest webinar is now available on demand! 🦋 In "Facilitating Patient Preference in Rare Epilepsy Studies", we asked our panel of experts from epihunter SynGAP Research Fund (SRF) for SYNGAP1-Related Disorders and Fundación Síndrome de Dravet how we should capture data to balance the needs of regulators seeking real-world data and the #epilepsy patients and caregivers who want to measure what matters to them. View the recording here 👇 https://hubs.li/Q02C_8Cr0 #eCOA #DigitalHealth