A Nature study led by Children's Hospital of Philadelphia’s Michael Hogarty, MD, has shown that in preclinical models, limiting certain amino acids through diet may enhance the effects of an FDA-approved drug for high-risk neuroblastoma. The findings lay the foundation for new strategies to treat high-risk neuroblastoma, the survival rate of which remains around 50%. “After many years of work in this area, it’s exciting to see this breakthrough that may lead to more effective therapies for children with neuroblastoma,” said Dr. Hogarty, who worked alongside collaborators at Princeton University and the University of Zurich. Read the full story on Cornerstone: http://ms.spr.ly/6043sQmCz
Study finds diet may boost neuroblastoma treatment
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Todd Harris, CEO of Tyra Biosciences, discusses the company’s experimental once-daily, oral medicine for achondroplasia; what’s known about it from studies conducted to date, and why he believes it will offer competitive advantages over existing therapies. Global Genes #RARECast https://lnkd.in/gd58cayr
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In a new episode of the #RARECast podcast by Global Genes, our CEO Todd Harris talks with host Daniel Levine about key factors that differentiate our investigational approach to support the management of #achondroplasia through selective #FGFR3 inhibition. Listen here for insights on our progress in research and drug discovery: https://lnkd.in/gzw3v8qt Tyra Biosciences #TyraBiosciences
Todd Harris, CEO of Tyra Biosciences, discusses the company’s experimental once-daily, oral medicine for achondroplasia; what’s known about it from studies conducted to date, and why he believes it will offer competitive advantages over existing therapies. Global Genes #RARECast https://lnkd.in/gd58cayr
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Todd Harris, CEO of Tyra Biosciences, discusses the company’s experimental once-daily, oral medicine for achondroplasia; what’s known about it from studies conducted to date, and why he believes it will offer competitive advantages over existing therapies. Global Genes #RARECast https://lnkd.in/gzw3v8qt
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ANGPTL4, a key regulator of triglyceride metabolism, is the focus of our latest video presentations from the 10th Closed Scientific Expert Meeting of the Triglyceride Forum Editorial Board. Find out why ANGPTL4 has become an important target for TG-lowering therapies and catch up on the latest results of preclinical and clinical studies of these novel agents: • ANGPTL4: New insights into an “old” lipid regulatory protein Sander Kersten, Wageningen University, The Netherlands 🔗 https://lnkd.in/eHPajy5H • ANGPTL4 inhibition: Phase 2a and ongoing trials Nicholas Marston, Brigham and Women’s Hospital, Boston, USA 🔗 https://lnkd.in/eR6dShbu #triglyceride #ANGPTL4 Wageningen University & Research European Atherosclerosis Society Brigham and Women's Hospital #lipidologists #cardiologists
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Hyperparathyroidism is a complex endocrine disorder where dysregulated Parathyroid Hormone (PTH) secretion disrupts the body's crucial calcium homeostasis, often due to a benign parathyroid adenoma. While surgical parathyroid removal remains the gold standard treatment, understanding the underlying molecular mechanisms is key to advancing future therapies. This requires robust and reliable research models. At AcceGen, we are committed to supporting this vital research. Our rigorously validated biological tools, including Human Parathyroid Cells and Disease-Specific PBMCs, provide scientists with the quality materials needed to study PTH regulation, calcium signaling pathways, and tumor biology with precision. Explore our resources to empower your next breakthrough in endocrine research. https://lnkd.in/gHv9VpnC #Hyperparathyroidism #EndocrineResearch #PTH #CalciumHomeostasis #MedicalResearch #Biotech #LifeSciences #Pharma #ParathyroidAdenoma #AcceGen
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Epigenetics Is Quietly Reshaping the Future of Healthcare The global epigenetics market is growing at an impressive pace: 2024 → $2.03 billion 2025 → $2.24 billion Forecast 2030 → $4.29 billion (CAGR 13.9%) 🚀 What’s driving this acceleration ? → Reduced sequencing costs and faster turnaround times → Expansion of population screening programs → Increasing demand for epigenetics-based therapeutics & diagnostics → Revolutionary technologies (NGS, single-cell analysis, advanced bioinformatics) 💡 Why is this important ? Epigenetics is no longer a niche research tool; it is becoming the cornerstone of personalized medicine, drug discovery, and diagnostics. Challenges remain (data complexity, reproducibility, regulatory frameworks), but the greatest opportunities lie in applications beyond cancer: neurodegenerative, autoimmune, and cardiovascular diseases. 📚 Resources to explore: NIH Epigenomics: https://lnkd.in/eHRzk6BC Nature – Epigenetics: https://lnkd.in/eFgVrdBV IHEC: https://lnkd.in/erSzqvF9 Epigenetics Market Report: https://lnkd.in/egqv8h-s The next decade will belong to innovators capable of transforming epigenetic knowledge into clinical reality. #Epigenetics #Genomics #HealthcareInnovation #PersonalizedMedicine #Biotech #Diagnostics #DrugDiscovery #Bioinformatics #HealthcareTransformation
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🌍 Today is #WorldSightDay. Globally, at least 2.2 billion people live with blindness or vision impairment. Cell therapy and regenerative medicine bring hope and several clinical trials are already underway. Yet, moving from lab to clinic requires strict clinical compliance and consistent production, both of which demand significant time and effort. Our full-length, human recombinant Biolaminin® proteins replicate the eye microenvironment in a defined, clinically relevant manner. They enable: Enhanced attachment and maturation of RPE cells in vivo-like functionality Improved barrier function and consistent calcium signaling, indicating stable, functional RPE cells Improved cell survival and functionality during and after bioprinting. 📩 Contact our expert team now and take your vision research further. #LoveYourEyes #RegenerativeMedicine #Celltherapy #BioLamina
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New Clinical Breakthrough in Autoimmune Therapy A new New England Journal of Medicine correspondence reports the first-in-human trial of in vivo CD19 CAR T-cell therapy for refractory systemic lupus erythematosus (SLE) Unlike traditional CAR T approaches that require complex ex vivo manufacturing, this study used lipid nanoparticles (LNPs) delivering CAR mRNA to reprogram patients’ T cells inside the body. Highlights: 1. Functional CAR T cells appeared within hours post-infusion 2. Rapid and sustained B-cell depletion was achieved 3. Patients showed improvements in autoantibody levels and disease activity 4. Encouraging safety profile — no severe neurotoxicity or grade 3–4 CRS This innovative “in vivo CAR T” strategy could make cellular therapies more scalable, cost-effective, and accessible, opening new possibilities for autoimmune diseases. Kudos to the authors: Qian Wang, Ze Xiu Xiao, Xu Zheng, Guihong Wang, Linlin Yang, Lina Shi, Nan Xiang, Xingbing Wang, Gao-Feng Zha, Georg Schett, and Zhu Chen, and their affiliates at: 1. First Affiliated Hospital, University of Science and Technology of China 2. Shenzhen MagicRNA Biotechnology 3. Anqing Municipal Hospital 4. Anhui University of Science and Technology 5. Friedrich-Alexander-University Erlangen–Nuremberg Read more: https://lnkd.in/gnfzcCPu #AutoimmuneDisease #Lupus #CARTcell #mRNA #LNP
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𝗔 𝗙𝗼𝘂𝗻𝗱𝗮𝘁𝗶𝗼𝗻𝗮𝗹 𝗕𝗿𝗲𝗮𝗸𝘁𝗵𝗿𝗼𝘂𝗴𝗵 𝗚𝘂𝗶𝗱𝗶𝗻𝗴 𝗩𝗶𝗮𝗰𝘂𝗿𝗲‘s 𝗠𝗶𝘀𝘀𝗶𝗼𝗻! 🚀 🔬 A landmark study published in Nature by our CSO, Dr. Heiko Lickert, unveiled the critical role of 𝗶𝗻𝗰𝗲𝗽𝘁𝗼𝗿, a novel inhibitor of insulin signaling in pancreatic β-cells. The study showed that in vivo genetic ablation of inceptor function significantly increases β-cell insulin sensitivity, proliferation, survival, and function, leading to improved glucose tolerance. This wasn't just a discovery; it was the identification and validation of a completely novel drug target for β-cell protection and regeneration for a causal therapy of diabetes. At 𝗩𝗶𝗮𝗰𝘂𝗿𝗲, we are building directly upon this breakthrough discovery. We are actively leveraging on deep mechanistic understanding of β-cell biology to develop innovative treatments aimed at sensitizing β-cells to insulin, offering a new drug intervention point to prevent β-cell failure and revolutionize diabetes care. 👉 If you want to delve deeper into this groundbreaking research, read the original paper here: https://lnkd.in/e54cRgw
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AMD prevention New intervention emerges in fight against age-related macular degeneration (#AMD). NIH-supported research reveals that boosting apolipoprotein M (ApoM) may correct cholesterol metabolism issues that contribute to retinal cell damage. Increasing ApoM in preclinical models slowed AMD progression, highlighting a new potential strategy to prevent or delay vision loss. For ophthalmologists and vision scientists, this insight offers a promising translational path beyond current late-stage therapies. Read more: https://brnw.ch/21wVMtP #NIHfunded #VisionResearch #VisionNews #ResearchNews #TranslationalScience #MacularDegeneration #AMDPrevention
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