UnitedHealthcare Children's Foundation’s Post

🌟 Empowering families with HeFH with HoFH knowledge! 🌟 Early diagnosis in HoFH can save more than one heart and life within a family. Detecting HoFH involves blood tests, physical exams, and reviewing family history. By identifying affected individuals early—especially those with high cholesterol or early signs of cardiovascular disease like heart attacks or strokes—we can potentially impact other family members. Genetic testing can also confirm HoFH. Beyond medical intervention, facing a life-threatening condition requires more. It requires unwavering support and solidarity. Every moment counts. Participate in our logo competition to stand with those battling HoFH and show them they're not alone in this fight. https://lnkd.in/eb7cy7Wi #Unite4HoFH #RareDisease, #UseHeart to #KnowHoFH and #FindHoFH #Maythe4thbewithyou

May is Cystic Fibrosis (CF) Awareness Month. The theme this year is recognizing the resilient spirit of the CF community. Nearly 40,000 people in the United States have CF, a rare genetic disease. CF can affect people of every racial and ethnic group. Learn more: https://lnkd.in/gU5ds5Rc #CFAwarenessMonth #CysticFibrosis

Are you a newly diagnosed liver-affected Alpha? Then register for the upcoming 2024 Alpha-1 Foundation National Conference to watch Dr. Teckman's presentation "Alpha-1 Antitrypsin Deficiency 101: Liver." To view the full agenda: https://lnkd.in/eDAtgspi

some new information about the FDA Operation Warp speed pilot, with 4 companies selected. Content from Endpoints, which I am a subscriber to. Companies listed below. I hope very much this works and accelerates progress! FDA officially selecting four participants to communicate more closely with: Denali Therapeutics, Larimar Therapeutics, Grace Science and Neurogene. One piece of info from the article: Grace Science, co-founded by Nobel winner Carolyn Bertozzi, said Monday that its GS-100, an AAV9-based gene therapy for NGLY1 Deficiency, a life-threatening rare disease with no approved therapy, was accepted into the pilot. Grace is running a Phase 1/2/3 dose-finding study to investigate the long-term safety, tolerability, and efficacy of the gene therapy in those aged two to 18 with NGLY1 Deficiency.

Take a look at the re-vamped fibrosis page on PhenoVista's website here! https://hubs.ly/Q02sYTf20 There's a general overview of our capabilities in this disease area and a case study of a completed client project. If you don't find what you're looking for or want to discuss challenges you're facing in fibrosis R&D, shoot let's chat!

Today, on World Hemophilia Day, learn more about how Specialty Therapeutic Care, our hemophilia center of excellence, supports people living with this rare genetic bleeding disorder: https://bit.ly/440BuEq #STC #hemophilia #centerofexcellence

Join the Anovo team as we observe World Homocystinuria Awareness Day, standing in solidarity with those affected by this rare metabolic disorder. Homocystinuria (HCU) is a rare genetic condition that affects approximately 1 in 200,000 individuals worldwide. It's caused by the body's inability to process the amino acid methionine, leading to the accumulation of harmful substances in the blood and urine. This can result in serious health issues such as heart disease, vision problems, and intellectual disabilities if left untreated.   Anovo is proud to support patients and families who rely on specialty and orphan drugs to treat HCU. Our partnership with Recordati Rare Diseases North America enables us to bring a promising treatment to market that helps reduce homocysteine levels in the blood, improving the lives of those with this rare condition. Let’s shine a light on World Homocystinuria Awareness Day and show our commitment to making a difference in the lives of HCU patients and their caregivers. #HomocystinuriaAwareness #RareDisease

Our most advanced pipeline program got further strengthened today with the receipt of the European #OrphanDrug Designation to treat Complex Regional Pain Syndrome. ⬇ Chronic pain remains a global public health problem. Synerkine aims to contribute a unique pain solution based on our profound understanding of cytokine biology and the crucial role of persistent inflammation in chronic pain. Receiving the orphan drug designation for our most advanced development program in #CRPS adds another valuable building block to our commercial strategy and validates the quality of the data package generated so far. Want to learn more? Meet Synerkine CEO Hans Preusting during the upcoming #BIOEurope partnering conference in #Stockholm, November 4-6th. #NonOpioid #ChronicPain #CRPSAwareness #OpioidCrisis #PainAwarenessMonth

May is Cystic Fibrosis Month. “Every May, the cystic fibrosis community comes together for CF Awareness Month. The theme of this year’s CF Awareness Month is, “Resilient.” Learn more about CF and how you can help this resilient community! https://lnkd.in/eGuAGitM

Don’t miss this blog by my team member, and good friend Derek Ansel, MS, CCRA, which delves into the future of rare disease clinical trials! #rarediseaseresearch #geneticcounseling #genetictesting