Welcome to NORD
News and Events
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Why Rare Disease Research is on the Upswing Despite reductions in R&D spending, research on rare diseases is going full-steam ahead. Read Why > |
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Read NORD Testimony From Congressional Hearing NORD VP Diane Dorman was one of eight witnesses, and the only patient representative, invited to testify at PDUFA hearing. Read NORD's testimony > |
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FDA Approves Drug For Rare Form of Cystic Fibrosis The FDA has approved a new treatment for a rare form of CF. Read blog by FDA official on the collaboration that led to this breakthrough result. |
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The New York Academy of Sciences Will host a conference on March 9: Complex Drugs in Light of Established Regulatory Guidance. Read More > |
News for Patients
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New UDP Applications Being Accepted The National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) reopened the application process on Dec 1. More > |
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NORD and EURORDIS Launch 16 Rare Disease Communities Ehlers-Danlos syndrome is the 15th disease to launch on the Rare Disease Communities site, an international online patient community. Visit the site and learn more about the project > |
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Study for Parents of Children with Rare Disorders A PhD student at Wurzweiler School of Social Work is conducting a study of parents of children with rare disorders who had a delayed diagnosis. The study involves a phone interview and a short questionnaire. More > |
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View the 2012 Rare Disease Day US Website This year, Rare Disease Day will be observed around the world on February 29, 2012. Learn more > |
News for Patient Organizations
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FDA 101 From NORD's Chief Medical Officer View lectures from Dr. Timothy Cote on why we need FDA and how it works. More > |
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Research Grants Announced for Myocarditis The Myocarditis Foundation announced the award of two research grants for 2012-2013. View the press release > |
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Member Organization Thanks Mexican President Calderon The Mexican government recently added language about "orphan diseases and drugs" to the health law. Our member Proyecto Pide un Deseo México wrote this letter in acknowledgment. Read the Letter > |
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The TS Alliance released a new white paper The paper is titled "Unlocking a Cure for Tuberous Sclerosis Complex: An Assessment of Scientific Progress and Research Needs". Read the press release > |
News for Medical Professionals
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Taubman Medical Research Institute Establishes $100,000 Annual Prize April 1 is the deadline for nominations for a new translational science prize to recognize a medical scientist for work to transform laboratory discoveries into clinical application. More > |
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Sanford-Burnham to Host Third Annual Rare Disease Day Symposium The event will focus on Identifying and Treating Genetic Diseases in Children. More > |
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NIH to Host Workshop in Bethesda, MD NIH is sponsoring a scientific workshop on Rare Syndromic Body Fat Disorders. Read More > |
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News for Industry
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Genetic Cause of Rare Immune Disease Identified A mutation in the PLCG2 gene that codes for an enzyme involved in the activation of immune cells has been found to be associated with a form of cold urticaria. Read more here> |
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Read draft guidance for industry on Humanitarian Use Device (HUD) posted Dec. 13 by FDA. More > |
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Overview of Drug Review in 2011 View recent presentation by John Jenkins, Director of the Office of New Drugs in FDA's CDER, providing overview of drug review in 2011. Read More > |
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Timothy R. Cote, MD, MPH, NORD's Chief Medical Officer, and Pat Furlong, a NORD Board member, will speak at the upcoming Rare Disease Leaders' Forum in San Francisco March 12-14. More > |
