Evox Therapeutics Ltd’s Post

Feb 29 is World Rare Disease Day. We are highlighting a few recent publications to help spread awareness of rare disease research. Factor (F)VIII prophylaxis or emicizumab is recommended for patients with severe Hemophilia A (HA), a rare genetic bleeding disorder. However, valoctocogene roxaparvovec gene treatment shows promising results. In this early cost-effectiveness assesment in the Netherlands, the novel gene therapy valoctocogene roxaparvovec compared to prophylactic FVIII and emicizumab was estimated to result in greater QALYs gains for less costs. Read the whole study here 👉 https://bit.ly/48jog6v #RareDiseaseDay #LightUpForRare #SpreadAwareness #ShareYourColours

Today on February 29th, the rarest day of the year, is #RareDiseaseDay. There are around 7,000 rare diseases. Each one affects only a small number of people but together they affect the lives of 3.5 million people in the UK and an estimated 300 million people globally. Not very rare at all and yet, the majority of these conditions lack specific treatments. Cell and gene therapies are gaining momentum across a growing number of therapeutic areas, including rare diseases. By addressing the root cause rather than merely treating symptoms, these therapies hold the promise of one-time treatments or even cures. At Broken String Biosciences we envision a future where cell and gene therapies are not only transformative but also safer, more precise and effective for patients. We proudly support Genetic Alliance UK and Rare Disease Day in raising awareness about the 350 million people living with rare diseases. To learn more and find out how you can get involved, please visit: rarediseaseday.org #RareDiseaseDay #GeneTherapies

Today is #RareDiseaseDay. 300 million people worldwide suffer form a rare disease. That's more than the population of England, France, Turkey, Kenya, and Peru combined. A rare disease diagnosis can be especially devastating; often for an uncommon disease there is less known about how they can be treated, fewer physicians with know-how about them, and fewer pharmaceuticals being developed to treat them. Some time ago, I watched the excellent documentary The Gene, by Ken Burns, and based on great book of the same name by Siddartha Mukherjee. In addition to showcasing the history of genetics it also profiled patients and physicians who are fighting to cure rare diseases through scientific breakthroughs. They included Dr. Wendy Chung, a Boston physician who has become an expert on rare genetic diseases including the terrible disease KIF-1A. They also included Audrey Winkelsas, a scientist who was born with a rare genetic disease called spinal muscular atrophy, and who is now pioneering research that may one day cure SMA. So on this day, it is my hope that as a society we can be inspired by the resilience of the patients and the ingenuity of the physicians, scientists, caregivers and innovators who are working to find cures and treatments for Rare Diseases. For more information, check out https://lnkd.in/eMpW6bgv #RareDiseaseDay2024

Today is Rare Disease Day. Did you know there are over 7000 rare diseases that affect over 300 million people worldwide? These diseases may individually impact a small percentage of the population, but together, they represent a significant health challenge. At CordenPharma we're actively contributing to the development and manufacture of many drugs for rare diseases such as Acute Myeloid Leukemia, Rett Syndrome, Cystic Fibrosis, Fragile X Syndrome, Gaucher Disease, Huntington’s Disease, and Idiopathic Pulmonary Fibrosis. Let's raise awareness and continue our efforts toward advanced treatments and finding cures. Together, we can make a difference. www.rarediseaseday.org 💙 #RareDiseaseDay #rarediseases #TogetherWeCan #CordenPharma #CDMO

Today is #RareDiseaseDay - on the rarest of all days! 72% of all rare disease are genetic, and our team at Genomenon is on a mission to make genomic information actionable for patients living with rare diseases and cancer. Join us in helping raise awareness of patients and their families living with a rare disease, and the organizations and researchers working to find cures! Living with any illness presents challenges, but rare diseases, affecting fewer than 1 in 2000 people, pose unique hurdles. Neglected, they suffer from a lack of medical expertise, limited understanding, and constrained research, making diagnosis and treatment arduous. Patients often endure years seeking diagnosis and accessing treatment, while research efforts encounter similar obstacles. This emphasizes the importance of raising awareness about rare diseases during events such as Rare Disease Day. Learn ways to get involved: https://lnkd.in/gk6hbAF #rarediseaseday2024 #rarediseaseawareness #shareyourrare #showyourcolors

This #RareDiseaseDay, TFS stands united in raising awareness and driving change for the 300 million people globally affected by rare diseases, along with their families and caregivers. Did you know that 1 in 5 #cancers are considered rare diseases? The realm of rare diseases extends through a broad spectrum of genetic disorders, including conditions like #DuchenneMuscularDystrophy and #HuntingtonsDisease. Rare diseases also manifest in various neurological conditions, such as #ALS and certain types of #epilepsy, among many other countless indications. And this is just the start. With extensive expertise in many of these areas, we recognize the critical importance of increasing awareness and supporting research. We are proud to contribute to the global movement towards better health outcomes, increased funding for research, and a more inclusive society for all, leveraging our depth of experience to advance research and bring hope to millions. Join us and learn more at https://lnkd.in/gk6hbAF. #TogetherWeMakeADifference

Today, February 29, 2024, is Rare Disease Day. At XPose Therapeutics, we are excited about our efforts on discovering and developing novel therapeutics, from scratch, in a fragment-based drug discovery approach, for Glioblastoma Multiforme (GBM), a rare and aggressive cancer, with a survival time of only about 15 months, by targeting APE1. Our recent publication (https://lnkd.in/gF3biba3) shows how our lead compound, XPTx-0091, enhances the cytotoxicity of MMS in glioma cells. #oncology #therapeutics #drugdiscovery #structuralbiology #rarediseases #rarediseaseday #glioblastoma https://lnkd.in/gk6hbAF

Kazia Therapeutics Limited stands in solidarity with Rare Disease Day, a day designed to highlight the importance of support and further research for patients suffering a rare disease. DMG (diffuse midline glioma) and AT/RT (atypical teratoid/rhabdoid tumor) are two such diseases - both are aggressive forms of brain cancer for which treatment options are very limited. We hope #paxalisib will help bridge the gap in care for these brain cancer patients. Learn more about #RareDiseaseDay here: https://shorturl.at/flNP0

“Raising awareness and generating change for the 300 million people worldwide living with a #raredisease, their families and carers.” RareDiseaseDay.org, 2024 ❔ Did you know❓ 🧑 A disease is considered rare if it affects fewer than 1 in 2,000 people. 🌍 Over 300 million people worldwide are affected by a rare disease. 👶 70% of rare diseases start in childhood. 🔍 More than 6,000 rare diseases have been identified. 🧬 72% of rare diseases are genetic. 😷 1 in 5 diagnosed cancers are considered rare. Find out more: https://lnkd.in/gk6hbAF #rarediseaseday #patientrecruitment #clinicaltrials #innovativetrials

Immunocal and lung diseases have been a life-changing combination for Cathy, a real-life user sharing her story. Cathy, diagnosed with Interstitial Lung Disease, found hope through Immunocal, a natural health product with 40 years of clinical research backing at McGill University Medical Research Centre. This innovative product, known to raise critical molecules like glutathione, has significantly improved Cathy's breathing after years of struggle. Cathy initially took one pack of Immunocal daily for two years before increasing her intake to two packs per day, leading to noticeable improvements in her energy levels and breathlessness. Against the odds, Cathy now enters her 5th year on Immunocal, defying her fatal prognosis and proving the product's effectiveness. Her respirologist is astonished by her progress, emphasizing the remarkable impact of Immunocal and glutathione on her quality of life. Cathy's gratitude for this life-saving product shines through her testimony. Cathy's inspiring journey serves as a beacon of hope and resilience for individuals battling lung diseases. To learn more about Immunocal and its transformative benefits, visit www.immunotec.com/possom. #lunghealth #asthma #copdtreatment #immunesystemhealth #immunesupport #InterstitialLungDisease #Glutathione #GSH #breathingexercises #lungdiseaseawareness #immunocalregular