White Fund SA’s Post

From sci-fi to reality → 👁️ transplants to restore vision Advanced Research Projects Agency for Health (ARPA-H) awarded $47 million to the project Human Eye-allotransplantation Innovation Advancement (THEIA), led in part by USC Roski Eye Institute, to supercharge bringing eye transplantation forward to clinical trial.

July is Sarcoma awareness month. Sarcoma is a rare and challenging form of cancer that affects connective tissues like bones and muscles and often goes unnoticed until it reaches advanced stages. Sarcoma Awareness Month is a crucial time to shed light on this disease and advocate for better research and support for those affected. The psychological impact on sarcoma patients is profound and multifaceted. According to recent studies, patients often experience significant anxiety, depression, and social isolation due to the physical and emotional toll of the disease. The rarity of sarcoma adds to the burden, making it difficult for patients to find support and resources tailored to their unique needs.   At Euretos, we believe in the power of patient-led, data-driven research. By harnessing advanced AI technologies, we can accelerate the discovery of new therapies and improve the quality of life for sarcoma patients. Our platform enables researchers to analyze complex datasets, identify novel drug targets, and develop personalized therapies. Academic researchers get free access. This Sarcoma Awareness Month, let's emphasize the importance of translational research and the need for a holistic approach to patient care that includes psychological support and community resources. Together, we can make an impact. #SarcomaAwarenessMonth #SarcomaResearch #TranslationalResearch #AIinHealthcare #PatientCare #MentalHealth #DrugDiscovery #SupportSarcomaPatients #HealthcareInnovation www.euretos.com

My talk at the National Institute on Aging (NIA) workshop on dementia with Lewy bodies (DLB): https://lnkd.in/eTuah747. Workshop was on advances in understanding translational and clinical aspects of DLB. So, my talk is on what we learned from our clinical studies that informs on DLB pathogenesis and designing clinical trials in DLB. But, also a very good summary of everything we know about our drug, neflamapimod, and its potential to treat underlying disease process in DLB.

BREAKING NEWS A new medicine for DMD, funded by Joining Jack, Duchenne UK and The Duchenne Research Fund has become the first approved treatment in the UK for all patients with Duchenne! The National Institute for Health and Care Excellence (NICE) has approved vamorolone for use on the NHS to treat Duchenne muscular dystrophy (DMD). Vamorolone, also known by the brand name Agamree, will be available to treat people with DMD aged four years and older. This is a landmark day for Team JJ as we have nurtured the development of vamorolone for 10 years. Read the full story here: https://lnkd.in/ejbZJiFf

🎶 Unless you've been on a desert island, you've probably heard about Celine Dion's powerful performance at the Paris Olympic Games, her first in four years after battling stiff-person syndrome (SPS). Did you know that CAR T cells might offer hope for this challenging condition? 🌟 A recent case study demonstrated the successful use of anti-CD19 CAR T cells in treating a severe, treatment-refractory case of SPS. This innovative therapy led to significant improvements in mobility and symptom relief, showcasing a promising path forward for patients facing similar challenges. 𝐊𝐞𝐲 𝐓𝐚𝐤𝐞-𝐀𝐰𝐚𝐲𝐬: 📈 𝐒𝐢𝐠𝐧𝐢𝐟𝐢𝐜𝐚𝐧𝐭 𝐜𝐥𝐢𝐧𝐢𝐜𝐚𝐥 𝐢𝐦𝐩𝐫𝐨𝐯𝐞𝐦𝐞𝐧𝐭𝐬: The patient experienced a substantial reduction in stiffness and a dramatic increase in walking distance. 💊 𝐌𝐚𝐧𝐚𝐠𝐞𝐚𝐛𝐥𝐞 𝐬𝐚𝐟𝐞𝐭𝐲 𝐩𝐫𝐨𝐟𝐢𝐥𝐞: The treatment was well-tolerated, with only low-grade side effects that were effectively managed. 🔬 𝐅𝐮𝐭𝐮𝐫𝐞 𝐩𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥: These encouraging results support further exploration of CAR T cell therapy in treating SPS and other neuroimmunological disorders. #SPS #CelineDion #CARTCells #JO2024 #GeneTherapy #CellTherapy #Neuroimmunology #InnovativeTherapy #HealthcareInnovation Simon Faissner, Jeremias Motte , Melissa Sgodzai, MSc., Christian Geis, Aiden Haghikia, Dimitrios Mougiakakos, Dominic Borie, M.D., Ph.D., Roland Schroers and Ralf Gold Ruhr University Bochum, St. Josef-Hospital Klinikum der Ruhr-Universität Bochum, University of Jena, Otto-von-Guericke University Magdeburg, Kyverna Therapeutics, Universitätsklinikum Knappschaftskrankenhaus Bochum GmbH

"I am afraid we are stuck with PRN100 without funding to move forward with a clinical trial. It's very disappointing for us all, but we keep trying to find investors/company partners. It's so hard in a rare disease where the business case is unclear." The message I just received breaks my heart. Today, PRN100 represents the only hope we have to halt the progression of Creutzfeldt-Jakob disease (CJD). Research has been slowed down multiple times over the last two decades due to lack of funding. As CJD is a very rare disease, developing a cure would not be profitable. Consequently, patients are left to suffer and die in one of the most horrifying ways imaginable. Trust me on this one. The cost of manufacturing PRN100 treatment and conducting a pivotal clinical trial will run into many millions. To help fundraise to support research at the MRC Prion Unit at UCL ➜ https://lnkd.in/gBw9ewra #MedicalResearch #RareDiseases #CJD #PrionDiseases #Healthcare #ClinicalTrials #FundingNeeded #PatientAdvocacy #ResearchFunding #CreutzfeldtJakobDisease #PRN100 #UCL

We are excited to share that University of Colorado Anschutz Medical Campus researchers have been awarded up to $46 million for Advanced Research Projects Agency for Health (ARPA-H)'s THEA program, which aims to drive groundbreaking research in curing total blindness through human eye transplantation. CU Innovations is thrilled to be part of this transformative journey and learn more about how this funding could change the future of vision restoration. #cuinnovations #cuanschutz

Did you catch the #news and see who won the big award last night? Sage's #multiplesclerosis Journal, shared their recent #March2024 issue's #coverstory #award went to.... None other than #ccfbme Kunio Nakamura + team of Cleveland Clinic Lerner Research Institute researchers. 🏆 🏆 👏 👏 👏 #Congrats to Dr. Nakamura & the entire team on this #groundbreaking study, "Ibudilast reduces slowly enlarging lesions in progressive multiple sclerosis" (below). Your research has significantly advanced our understanding of #ms and its treatment, demonstrating that ibudilast can decrease the volume of slowly enlarging lesions in patients by 23% over 96 weeks. It also highlights the potential of ibudilast for future #MStherapies, and the value of using slowly enlarging lesions as an #imaging #biomarker for chronic active lesions. Stay tuned for further updates this #awardsseason as Cleveland Clinic Dept. of #biomedicalengineering continues to bring #innovationinhealthcare from bench to bedside. #bme #coverstorychronicles Cleveland Clinic Innovations #inthenewsmondays

I recently presented in this video the deeply concerning research studies that link exposure to degraded plastics to increases in cancer, neurodevelopmental disorders, stroke, and heart attack. We have to believe that change is possible. https://lnkd.in/ehbYBZSz #nanoplastics #humanhealth

Someday timely diagnosis for patients with extremely early onset #Huntingtonsdisease is going to be lifechanging. I am excited to share our article to bring awareness of the subset of patients that may benefit most from these emerging therapies. Thanks to my coauthors Jonathon Lutz, Khalida Liaquat, MS, CGC, Jyes A. Querubin, MD and Sat Dev Batish, PhD, FACMG, FAAN #GeneChat #QuestDiagnostics https://lnkd.in/gC7-RxCN