Pirouette Medical’s Post

Introducing, MetX. The very foundation of the Metrodora mission consists of bringing together patients, providers, and scientists. Each entity holds a key to unlocking the answers behind complex illnesses. We knew the silos of medicine were hindering advancement, so we set out to try something different. Disrupting an entire industry takes time our patients don’t have. Symptoms are often derailing their dreams in the prime of their lives. We knew we had to work hard and fast. Since opening our doors a year ago, our team has seen hundreds of patients, launched clinical trials, and now we are excited to announce MetX, funded by a grant from the Metrodora Foundation and our visionary donors. This is how we come together to accelerate research, treatment, and cures. Patients are empowered to become partners through Metrodora Connect, a digital health toolkit where sharing health records, questionnaires, and wearable device data means an active role in research. A biorepository will host specimens to advance genetic, antibody, and cellular treatments. While leveraging partnerships with other major medical research organizations, Metrodora Biomics will use these specimens to discover targeted drug therapies. The tools, people, and passion already exist. It’s time they unite. We invite you to get to know MetX and the possibilities for all to make a difference right now. #MetX #MetrodoraFoundation #MetrodoraInstitute #Metrodora #MedicalNews #Medical #Medicine #Healthcare #Research #ComplexIllness #LongCOVID #MECFS #POTS #Migraine #Sjogrens #EDS #MCAS #ChronicPain #Fibromyalgia #Endometriosis #FoodAllergy #IBS #IBD

C-Path's Cure Drug Repurposing Collaboratory, and the CURE ID team will be onsite at the International Drug Repurposing Conference 2024 (#iDR24): Bridging boundaries for Innovative Drug Repurposing this week where key leaders in drug repurposing will unite. Co-organized by REMEDi4ALL, Beacon for Rare Diseases and MeRIT, the conference showcases how patient communities, researchers, industry and health authorities can benefit from cutting-edge drug repurposing. The agenda follows three topical paths in parallel: · Cancer · Rare Disease · Methodologies Find out more about the speakers, location, details here: https://lnkd.in/eRtYcAmZ Smith Heavner, PhD, RN | Marco Schito | Heather Stone | Shira Strongin | Keyla Tumas #CPath #REMEDi4ALL #Beacon #MeRIT #drugrepurposing #patientadvocacy

CDRC, Critical Path Institute (C-Path), and CURE ID team members will be traveling to Barcelona to attend the International Drug Repurposing Conference 2024 (#iDR24): Bridging boundaries for Innovative Drug Repurposing. Join them on 6-7 March 2024 at Sant Pau Art Nouveau World Heritage Site Sant for #iDR24, uniting key players in drug repurposing. Co-organized by REMEDi4ALL Consortium, Beacon for Rare Diseases, and MeRIT, the conference showcases how patient communities, researchers, industry, and health authorities can benefit from cutting-edge drug repurposing. The agenda follows 3 topical paths in parallel: · Cancer path · Rare diseases path · Methodologies path Explore cutting-edge insights, discuss challenges, and engage with experts. Find out more about the speakers, location, ticket prices and more here: https://lnkd.in/eRtYcAmZ Smith Heavner, PhD, RN| Marco Schito| Heather Stone| Shira Strongin| Keyla Tumas| REMEDi4ALL| Beacon for Rare Diseases| #REMEDi4ALL #Beacon #MeRIT #drugrepurposing #patientadvocacy

A nice viewpoint, describing opportunities and challenges associated with Project Optimus. We know that the model, developed years ago in the chemotherapy era, is no longer applicable to the drugs we are developing today: more is not (necessarily) better. In addition, most of the drugs developed today are given either continuously or for a long period of time: in this context, late onset/chronic toxicities and acute toxicities are equally important. We must now work on how we can incorporate this dose-optimization phase into clinical development, without loosing (too much) time. Collaboration with #fda and other Health Authorities will be very important. And I strongly believe that Patients and Patient Advocacy Groups should have a greater role in the design and analysis of the studies. https://lnkd.in/ed3Cri9h

What challenges does the advanced therapies sector face? Over the three days of Advanced Therapies Week (ATW) 2024 in Miami, Florida, US, 16-19 January 2024, DDW’s Megan Thomas heard from attendees about the obstacles the sector needs to overcome to succeed. https://lnkd.in/dev8gwmq #AdvancedTherapies #AdvancedTherapiesWeek #LifeSciences

🔬 Exciting findings from the ENVISION trial: UBX1325 shows promise in advanced #wetAMD cases. Despite not meeting primary endpoint, it's safe and well-tolerated. Dr. raj maturi shares insights on its unique mechanism targeting senescent cells. Significant proportion of patients didn't require anti-VEGF treatment through 24 weeks. Secondary analysis hints at efficacy in longer disease duration patients. Ongoing exploration of combination therapy potential in Part B of ENVISION study. 👉 Learn more in this article from Healio by Michela Cimberle, fact checked by Christine Klimanskis #agerelatedmaculardegeneration #maculardegeneration #cataract #ophthalmology #ophthalmologynews

Read the blog: https://ow.ly/CQpx50Sw2Ww Check out the latest AMCP Dossier Format Version 5.0. Learn about health disparities, real-world evidence, digital therapeutics, and more. Authors: Joshua Gauci, Irene Zahirovic (Zelner), PhD, Deepika Thakur #AMCP #HealthDisparities #RWE #DigitalTheraeutics

On #LimbGirdleAwarenessDay, we’re proud to announce the launch of C-Path’s new task force dedicated to advancing drug development for limb-girdle muscular dystrophies. Under the Rare Disease Cures Accelerator-Data and Analytics Platform, this collaborative effort unites experts from leading organizations to tackle the unmet medical needs in the LGMD community. This task force includes pivotal members: ML Bio Solutions (BridgeBio), Coalition to Cure Calpain 3, CURE LGMD2I FOUNDATION, Jain Foundation Inc, LGMD2D Foundation, LGMD2i Research Fund and The Speak Foundation. Together, we’re leveraging patient-level data, shared knowledge, and a neutral platform to expedite therapeutic advancements for those affected by LGMDs. As we work toward accelerating meaningful progress, our commitment remains to ensure patient voices and data drive innovation. Read the full details, here: https://lnkd.in/gFiffMB3 Alexandre Bétourné, PhD, PharmD, PMP Heidi Grabenstatter Douglas Sproule Kat Bryant Knudson Ramona Belfiore-Oshan Rachel DeConti Adora Ndu, PharmD, JD Anna Wade Cybele Gouverneur Jean-Pierre Laurent Jennifer Levy Jessica Evans Kelly Brazzo Laura Rufibach Patrick Moeschen #CPath #LimbGirdleMuscularDystrophy #RareDiseaseResearch #DataCollaboration #DrugDevelopment #DataSharing #RDCADAP #PatientVoice #Collaboration

Avidity Biosciences, Inc. has moved forward with a Phase 2 study of AOC 1044, which consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with phosphorodiamidate morpholino oligomers targeting exon 44. The study, which is enrolling by invitation, is designed to evaluate the pharmacodynamic effect of AOC 1044 on dystrophin protein production in skeletal muscle, as well as the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044, in Duchenne muscular dystrophy participants with mutations amenable to exon44 skipping. #mabs https://lnkd.in/eCpDiG49

Samuel Whitaker Whitaker and Jason Dong, co-founders of Mural Health, recently shared their predictions with PharmaVoice on how they believe the industry will develop in 2024. “We hope to see a heightened focus in the industry on removing financial barriers to clinical trial participation — barriers that disproportionately affect people from lower socioeconomic and minority groups and result in a lack of diversity that threatens the efficacy of the drug development process.” To read more, including predictions from other industry leaders, click here: https://ow.ly/xYIn50QrOLh #ClinicalTrialResearch #PatientAdvocates