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Trusted Resources: News & Events
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Sancilio Pharmaceuticals Company announces Altemia™ receives orphan drug designation from the European Medicines Agency for the treatment of pediatric patients with Sickle Cell Disease (SCD)
Sancilio Pharmaceuticals Company, Inc. (SPCI) today announced that the European Commission has granted Orphan Designation in the European Union to Altemia, the Company’s novel oral formulation being developed for the treatment of SCD in pediatric patients. This approval follows a positive opinion in February 2018 from the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).
Orphan Designation is granted by the European Commission to drugs that are intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating rare diseases. Rare diseases are those defined as having a prevalence of not more than five per 10,000 population in the European Union. The designation potentially qualifies the sponsor for ten years of marketing exclusivity upon approval, as well as fee reduction for various centralized activities, including applications for marketing authorization, inspections and protocol assistance.
news & eventsMaxCyte, Inc. to Present Positive Preclinical Data for Sickle Cell DiseaseMaxCyte, a US-based global company dedic...
videos & visualsTreatment Advances in Sickle Cell Disease, Amber M. Yates, MDhttps://www.youtube.com/watch?v=oFIOKC4r...
news & eventsNew Method of Creating Healthy Stem Cells Could Potentially Improve Treatment of Sickle Cell AnemiaResearchers at New York’s Weill Cornel...
videos & visualsGene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
education & researchPediatric Residents’ Perceived Barriers to Opioid Use in Sickle Cell Disease Pain ManagementObjective: Current guidelines recommend...
videos & visualsSickle Cell Breakthrough: Shauna’s Storyhttps://www.youtube.com/watch?v=Q-kXWV-s...
education & researchSevere loss of circulating dendritic cell subsets in splenectomised children with sickle cell diseaseSplenectomy is the mainstay of long-term...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.