Zevra Therapeutics’ Post

It is with overwhelming joy and a profound sense of hope that we share a groundbreaking announcement with you today. After years of tireless research, unyielding determination, and unwavering support from our incredible community, the FDA has approved the very first therapy for the treatment of Niemann-Pick Type C (NPC) disease. This newly approved therapy offers not just a treatment but a promise—a promise of improved quality of life, a promise of more tomorrows, and a promise that we are moving ever closer to a world where NPC no longer dictates the futures of our loved ones. It stands as a testament to what we can achieve when we come together with a shared purpose and a common goal. This is a day we will never forget—a day that redefines hope for all of us. Let us cherish this moment, honor the journey that brought us here, and look forward to a future where NPC is defeated. https://lnkd.in/gfMQMSE9

🌟 New Hope for NP-C Families! 🌟 Exciting news for the NP-C community! The FDA has approved Miplyffa, the first treatment for Niemann-Pick disease type C. This breakthrough offers a promising future for our loved ones. Learn more about what this means for NP-C families in Australia and how we can support each other. 💙 Read the full article here: https://wix.to/Lyfb2hl #NiemannPickDisease #NPC #Miplyffa #Hope #ANPDF #RareDisease #FamilySupport #Advocacy

🌎✨ The sixth annual #WorldNarcolepsyDay is hitting the Americas! September 22, 2024 is a day dedicated to raising awareness of #narcolepsy on a GLOBAL scale. Narcolepsy affects 1 in every 2,000 people, yet its awareness remains obscured by misconceptions and stigma. But together, we've been changing that, one year at a time! 🏅 Today, we honor the incredible community that stands strong in the face of this challenging condition. You inspire us daily with your resilience, boundless strength, and radiant positivity. Remember, you are never alone in this journey. Your story matters. YOU matter! Together, we rise, shine, and nap as needed! 💙 Thank you to our amazing partners and collaborators across the US and around the world who are helping to co-lead this effort. We cannot wait to see what everyone is up to today (share photos and use the hashtag #WorldNarcolepsyDay and tag Project Sleep)! ⏰ It's not too late to get involved! Check out all of our #WorldNarcolepsyDay2024 education & awareness tools: https://lnkd.in/gtkYSSAe