PPT-Rare Action network

Presentation by NORD June 16 2014 NORD Leading rare disease patient advocacy organization for gt 30 years Principal resource for federal agencies and corporations when addressing questions or issues concerning the rare disease community . “. 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. CampaignTracker. A brief overview of its design and function and what we have learned so far.. Background & Purpose. The . CampaignTracker. is a tool built to house Rare’s reporting tools and processes that provide a consistent global language with which to measure and assess the progress and quality of Rare’s work around the world.. Batten Disease. June 29-30, 2010 . FDA Public Meeting. “Considerations regarding the review and . regulation of articles for treatment of rare diseases”. Tracy VanHoutan, . Board Member . of the Batten Disease Support and Research Association (BDSRA). animals and plants.. Done by . Serega. . Potyakaylo. , . grade 6. How Are Rare Plants Conserved?. Botanists, ecologists and other resource specialists have many tools to conserve, protect, and manage these rare jewels of our natural heritage. Some of the most important and effective tools available are habitat conservation, off-site (ex situ) conservation, and law enforcement. Specialists and volunteers are essential to success in all aspects of any rare plant programs.. Gil . McVean. There are no new questions in population genetics. …only new types of data. Genome sequences of entire populations. Data with linkage to deep phenotype information. Data from multiple species. Andrew Morris. Advanced Topics in GWAS. Toronto, 30 May 2012. Introduction. GWAS have been successful in detecting . novel loci for complex traits:. typically characterised by common variants of modest effect;. Anthony Cosimano. Elements on the lower periodic table found in the earth’s crust. A few of these elements are used heavily in the production of technologically advanced goods. Such as.... Smart phones. f. rom Methodology to Practice. a. nd Back. Paul . Embrechts. Department of Mathematics . Director of . RiskLab. , ETH Zurich . Senior SFI Chair . www.math.ethz.ch/~embrechts. Summary:. A bit of history. 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. in the United States. Paul Melmeyer. Associate. . Director. of Public Policy. National . Organization. for Rare . Disorders. (NORD). Where. . W. e. . W. ere. What. . We. . H. ave . Accomplished. and Their Carriers . Using Compressed Se(. que. ). nsing. Or . Zuk. Broad . Institute of MIT and . Harvard. orzuk@broadinstitute.org. In collaboration with: . Amnon. Amir. Dept. . of Physics of Complex Systems, Weizmann . Page 53 of 60 Ralf‐Dieter Hilgers*, Franz KönigDepartment of Medical Statistics, RWTH Universit Aachen, Pauwelstr 30, D‐ 52074 Aachen, Germany Dr. RalfDieter Hilgers, Department of Medical Statis the inherited forms of the disease have an incidence of approximately 1 to 2: 400,000 [e ADCL is caused by mutations in structural genes coding for elastin (ELNFBLN5) and Aldehyde Dehydrogenase 18 Fa Will these high-tech industry elements continue in short supply?. By Keith R. Long, Mineral Resource Analysts. Western Mineral and Environmental Resource Science Center. Tucson, AZ. World Production of Rare Earths as Oxides 2009.