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
关于我们
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.
- 网站
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https://www.zevra.com
Zevra Therapeutics的外部链接
- 所属行业
- 制药业
- 规模
- 11-50 人
- 总部
- Celebration，FL
- 类型
- 上市公司
- 创立
- 2006
地点
Zevra Therapeutics员工
动态
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Yesterday, Zevra Therapeutics announced U.S. commercial availability of their product for the treatment of Niemann-Pick disease type C: https://bit.ly/4eKCFfc #NPC #NiemannPick
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For U.S. Healthcare Professionals Only: We are proud to share that MIPLYFFA (arimoclomol) is now available in the U.S. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. Enroll now to get started! https://bit.ly/3AExbVj #NPC #RareDisease See the Important Safety Information for MIPLYFFA (https://bit.ly/4eIUXNE) and Prescribing Information, including Instructions for Use (https://bit.ly/4fs3m9n).
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Zevra Therapeutics is looking forward to hosting an exhibit at the 10th Annual Genetic Rare and Immune Disorders Symposium (GRIDS), November 24-25, at the Capital One Hall in Tysons, Virginia. More information here: https://bit.ly/3OfRADr #RareDisease
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LaDuane Clifton, Zevra Therapeutics’ Chief Financial Officer, is participating in the BioFlorida, Inc. panel, “Florida Innovation: Redefining the Treatment Paradigms for Rare Diseases," on November 19, at 12:20 p.m. ET at the Walt Disney World Swan in Lake Buena Vista, Florida. Join us as we explore innovative approaches to treating #RareDiseases. Learn more about the event here: https://bit.ly/40TaG9C #BioFlorida #HealthcareInnovation
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#DidYouKnow? Hyperammonemia can cause a persistent bitter taste in your mouth, nausea, restless leg syndrome, headaches, and balance issues. Know the signs thanks to Zevra Therapeutics' and the National Urea Cycle Disorders Foundation's #CheckAmmonia resources: https://bit.ly/3BOeoXI #highammonialevels #UreaCycleDisorders #UCDs
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Zevra Therapeutics would like to extend a special thanks to the Burgos and Andrews families who shared their #NPC journeys and the important work of the Firefly Fund at our annual Zevra Zeal town hall meeting last month. We appreciate your continued support of our mission of developing treatments for #RareDiseases. #NiemannPick
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Zevra Therapeutics' AmplifyAssist is a comprehensive support program designed for caregivers and individuals affected by #NiemannPick disease type C and certain urea cycle disorders. Explore our resources for clinical education, insurance coverage assistance, access support and prescription fulfillment. #NPC #UCDs #RareDisease
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Zevra Therapeutics is committed to developing treatments for rare diseases. Our phase 3 clinical research study tests celiprolol, an investigational medicine that is being evaluated to determine its efficacy in treating Vascular Ehlers Danlos Syndrome (#VEDS). Learn more about the Decentralized Study Evaluating Celiprolol on VEDS Related Events (DiSCOVER) clinical trial and enroll here: https://bit.ly/3C305i8 #ClinicalResearch #RareDisease
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Zevra Therapeutics was pleased to present at the 2024 American Society of Human Genetics last week. Thanks to the organizers and all the participants for a great conference. We look forward to next year! #NPC #raredisease
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