The FDA approved the first treatment for Niemann-Pick disease type C. “Today's FDA approval marks a significant milestone for people living with NPC, as it represents the first treatment available for this devastating condition. We are immensely grateful for the unwavering support of the NPC community throughout this journey," said Neil F. McFarlane, President and CEO. Read the full announcement here: https://bit.ly/3TziV6g #NiemannPick #NPC
Zevra Therapeutics
Pharmaceutical Manufacturing
Celebration, FL 10,367 followers
A Rare Approach to Therapeutics
About us
We are a rare disease therapeutics company leading with science to make life-changing therapeutics available to patients with significant unmet needs. We involve key thought leaders, physicians, patients, care partners, and advocacy groups in all of our clinical and regulatory development strategies. With a keen understanding that drug development often requires creative solutions, we have the insight and expertise to forge new pathways to success that others have missed. By following the data without bias, our transparent narratives and common-sense perspective have successfully overcome complex development challenges to make much-needed therapies available to patients. Nimble and dauntless, we push boundaries beyond what is thought to be possible and advance new therapies that have the potential to bring meaningful improvement to patients’ lives.
- Website
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http://www.zevra.com
External link for Zevra Therapeutics
- Industry
- Pharmaceutical Manufacturing
- Company size
- 11-50 employees
- Headquarters
- Celebration, FL
- Type
- Public Company
- Founded
- 2006
Locations
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Primary
1180 Celebration Boulevard
Celebration, FL 34747, US
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2500 Crosspark Road
Coralville, IA 52241, US
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2200 Kraft Drive
Blacksburg , VA 24060, US
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1001 Pennsylvania Ave, NE
13th Floor North
Waashington, DC 20004, US
Employees at Zevra Therapeutics
Updates
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We are grateful to the healthcare providers and caregivers devoted to caring for patients living with #NiemannPick disease type C. Zevra Therapeutics thanks you for your hard work and support! https://bit.ly/3XW7WVY #NPC #NiemannPickDiseaseAwareness
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Zevra Therapeutics is proud to recognize Niemann Pick Awareness Month this October. Those affected by #NPC will share their stories in an effort to raise awareness of this devastating disease. We can join NATIONAL NIEMANN-PICK DISEASE FOUNDATION and help more people learn about the critical need for support, treatment and a cure. Please join us in building #NiemannPickAwareness by visiting: https://bit.ly/4eSxLgK Firefly Fund, Hope For Marian, AbbyStrong Fights NPC, Dana's Angels Research Trust, Australian NPC Disease Foundation Inc, Niemann-Pick UK (NPUK), International Niemann-Pick Disease Registry (INPDR), International Niemann-Pick Disease Alliance (INPDA)
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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
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🌟 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
A New Hope for NP-C Families: Introducing Miplyffa
npcd.org.au
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🌎✨ 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
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Zevra Therapeutics reposted this
Best news #fireflyfund
Such an emotional and exciting day for the #NPC Community. Thank you to the #FDA for giving our children a lifeline. With this approval the entire rare disease community can bear witness to a demonstration by the #FDA that they are willing and ready AND equipped to use regulatory flexibility to approve drugs for the rare disease community. #NPC families have been waiting decades for this moment and Chris Andrews and I are elated to add #Arimoclomol (Mypliffa) to our therapeutic arsenal for Belle & Abby to have the best chance of rendering NPC a chronic, instead of fatal, disease. What a day! While the drug approved today helps #NPC patients, the WIN today helps the entire rare disease community. #fireflyfund #multipledrugs #combinationtherapy
FDA Approves First Treatment for Niemann-Pick Disease, Type C
fda.gov
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October is Vascular Ehlers-Danlos Syndrome (#VEDS) Action Month--a great time to support those living with this #raredisease that impacts connective tissue throughout the body. Learn how you can play your part in #chargingforward4VEDS with The VEDS Movement: https://bit.ly/47PV4Fx
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Zevra Therapeutics is proud to sponsor the upcoming 2024 Connecting Families UCD Foundation Meetup in San Diego October 4-5. Find more details on the event here: https://bit.ly/4dOW9j5 #UreaCycleDisorders #UCDs
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Zevra Therapeutics is pleased to sponsor the 10th Annual Cure the Cycle Challenge on October 5. Join Team Zevra and the National Urea Cycle Disorders Foundation in celebrating the life and impact of Jim Stavas as we work toward a cure for #UreaCycleDisorders. Learn more, register, and donate to the cause here: https://bit.ly/3B5xMio #UCDs