PPT-RARE DISEASES AND THE EUROPEAN HEALTH DATA SPACE

Juan Fernando Muñoz General Secretary for Digital Health Information and Innovation New features introduced Field Natural persons Rights Obligations of States Entities. “. EMERGING THERAPIES FOR RARE DISEASES. ”. Emil D. Kakkis, M.D., Ph.D. .. President and . Founder. CENTER FOR ORPHAN DISEASE RESEARCH . AND . THERAPY SYMPOSIUM. FRIDAY . MAY 2, 2014. Prafulla Dawadi. Topics in Machine Learning. Outline. Part I. Examples. Rare Class, Imbalanced Class, Outliers. Part II. (Rare)Category Detection. Part III. Kernel Density Estimation . Mean Shift and Hierarchal Mean Shift. DISEASES. “ARE WE READY FOR THE CHANGE THAT WE NEED TO FIND EFFECTIVE TREATMENTS FOR ORPHAN DISEASE?”. 2. YES!. Rare disease is very ‘popular’!. Rare disease <200,000 Americans have disease. Agency’s Comet Landing. November 12, 2014:. First-Ever Soft Landing on a Comet. (Comet 67P/. Churyumov-Gerasimenko. ). The Rosetta Mission. Launched in 2004: 10 Years in the Making. Graphic: . Time. Prafulla Dawadi. Topics in Machine Learning. Outline. Part I. Examples. Rare Class, Imbalanced Class, Outliers. Part II. (Rare)Category Detection. Part III. Kernel Density Estimation . Mean Shift and Hierarchal Mean Shift. Rare Earth Element . Challenges. Roughly 87% of REEs came from China in 2014. Potential national security and supply risk for critical rare earths for defense and clean energy. Y, . Nd. , . Eu. , . Dy. “2. nd. World Congress on Rare Diseases and Orphan Drugs”. Date: 29. th. and 30. th. June, 2017. “I am . Dr.. Gayathri Balasubramanian, a part of focus scientific research . center. , the scientific arm of . Issues in Clinical Trial Design for Rare Diseases. Jonathan C. Goldsmith, MD, FACP. Associate Director Rare Diseases Program/Office of New Drugs. Center for Drug Evaluation and Research. FDA. November 7, 2016. European. perspective. EUCARE session, Porto, August 27th, 2010. Michel . Baulac. , Paris. ILAE . Vice-President. The . European. Scope. EUCARE = Joint . Task. Force . from. ILAE and IBE. European. inclusion 1 6.19 Rare diseases See Background Paper 6.19 (BP6_19Rare.pdf) Background In the EU, a disease is considered to be rare when the number of people affected is less than 5 per 10 000. There 1 / 3 ‘ New scientific paper confi rms 300 m illion people living with a rare disease worldwide Paris, 17 September 2019 - A new scientific paper confirms the number of people living with a rar Rare Disease DayFrequently Asked QuestionsWhat is a rare disease The most complete listing rarediseasesinfonihgov/diseasesWho is affected by rare diseasesManybut not allrare diseases are genetic Some | Version 02 | March 2020 : Orphanet nomenclature and classification of rare diseases www.orpha.net www.orphadata.org Procedural document on the Orphanet nomenclature and classification of rare . to. . care. and . on. European . cooperation. EESC . public. . hearing. :. Ensuring. . strong. European . solidarity. . for. . rare. . disease. . patients. Prof. Dr. . Ildiko. . Horvath.