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News and Press Releases

News

28 January 2021

Putting Rare Diseases Patients First!® is pleased to announce that Sickle Cell Disease – The New Frontier Part 2 will take place on 28 May 2021. This Webinar will be a continuation of the very successful Webinar that was held on 20 May 2020.  We are pleased to announce that Beverley Francis-Gibson of Sickle Cell Disease Association of America will be participating as our special guest. Find out more and register at www.puttingrarediseasespatientsfirst.org/sicklecell

Press Releases

Putting Rare Diseases Patients First!® – Putting Rare Diseases Patients First!® announces today that it has submitted a Citizen’s Petition to the FDA to add Sickle Cell Disease to FDA’s Tropical Disease Priority Review List. Dr. Lorna Speid, Founder and President of Putting Rare Diseases Patients First!® explained that Sickle Cell Disease is a rare disease in the United States, where it afflicts a majority African American population. However, outside  of the United States, particularly in the developing world, the disease primarily afflicts patients of African descent, many of whom are marginalized, and who lack the resources to access the latest treatments for Sickle Cell Disease. They certainly lack the resources to develop novel treatments for their disease.  In the developing world, many children afflicted by this disease, many of whom will not see their fifth birthday. Dr. Speid provides evidence in the Citizen’s Petition to support the qualification of Sickle Cell Disease for addition to the Tropical Disease PRV List.  Dr. Speid further explains that PRDPF!’s goal in submitting the Citizen’s Petition has been to provide FDA with the evidence needed to rectify address the situation. The Docket can be found at regulations.gov by searching for fda-2020-P-167