Welcome to NORD
News and Events
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NORD VP Participates in White House Event on Innovation in Drug Development VP of Public Policy Diane Dorman was an invited guest at an event marking the release of a major new report by the President’s Council of Advisors on Science and Technology. More > |
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Which Orphans Will Find a Home? A NORD medical adviser has responded to a Hastings Center report on resource allocation and the cost of providing treatment to patients with rare diseases. Read the original report. Read the response by Doris T. Zallen, PhD. |
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First-Ever Treatment for Progeria Lonafarnib, a type of farnesyltransferase inhibitor originally developed to treat cancer, has been shown to be an effective treatment for progeria. Read more > |
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NORD Supports Pediatric Research Act NORD has written to the House & Senate sponsors of the National Pediatric Research Act, pledging support but urging coordination with existing research network. Read the statements> |
News for Patients
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Rare Disease Day is February 28, 2013! NORD is gearing up for Rare Disease Day 2013! Make sure you are signed up as an ambassador to get the updates when we launch the campaign. More > |
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A guide for young people with disabilities transitioning to adulthood has been developed by the California Health Incentives Improvement Project. More > |
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The first study to be conducted by NeuroNext is a Biomaker and Natural History Study in infants with Type I SMA. More > |
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Improved Gene Therapy Restores Immune Function for Some with SCID A study has demonstrated an optimal gene therapy approach for these children that included the use of a specific retroviral vector. Learn more > |
News for Patient Organizations
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A Board Member from a NORD Member Wins Award AA&MDSIF Medical Advisory Board member, Dr. Neal Young, has been awarded the 2012 Samuel J. Heyman Service to America Science and Environment medal. More> |
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NORD Organizational Membership Meeting Held in Philadelphia Members participated in NORD’s Regional Membership Meeting in Philadelphia on Friday, September 21st. View Images > |
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FDAVoice Blog on Protections for Children in Clinical Research This post discusses expanded access regulations and other considerations for parents whose child has a life-threatening disease for which there is no approved treatment. Read more > |
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Genzyme Announces Winners of 2012 Patient Advocacy Leadership Awards This grant program supports outreach programs by non-profits that work on behalf of patients living with lysosomal storage disorders. Read more > |
News for Medical Professionals
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ENCODE Project Reveals that Non-Coding DNA has a Function Numerous papers now suggest that many diseases may be caused by alterations in how genes are turned on, rather than mutations in genes that code for specific proteins. More> |
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New Guideline on Diagnosis of Creutzfeldt-Jakob Disease The American Academy of Neurology has published a new guideline focused on the diagnosis of sporadic Creutzfeldt-Jakob disease. More > |
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NORD Research Grant Recipient Wins Award Dr. Anthony Shum, a 2010 NORD research grant recipient, has won the prestigious American Thoracic Society Foundation/Pulmonary Fibrosis Foundation Research Award. More > |
Tenure-Track Faculty Position in Rare Diseases The Dept. of Biological Sciences & the Dept. of Chemistry and Biochemistry at the University of Notre Dame invite applications for an Assistant Professor level position in rare diseases. More > |
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News for Industry
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NORD Board Members Honored by PharmaVoice Two members of NORD's Board of Directors have been named to the "PharmaVoice 100". Read more > |
NORD Commends PhRMA for Adopting Principles for Interaction With Patient Organizations The guidelines emphasize respecting the values and independence of patient organizations. Read the guidelines > |
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FDA-NIH Science of Small Clinical Trials This course will present an overall framework and provide training in the scientific aspects of designing and analyzing clinical trials based on small study populations. Read more> |
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NORD's President Interviewed about Rare Disease R&D NORD President Peter L. Saltonstall was interviewed recently by PharmaPhoroum. Read the article > |
