Welcome to NORD
News and Events
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FDA and EMA to Co-Host Designation Workshop FDA OOPD and its European counterpart will host an Orphan Designation and Grant workshop in Silver Spring, MD, on Oct. 12. Learn more about the meeting > |
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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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Christopher Austin to Serve as 1st NCATS Director NIH Director Francis Collins has announced that Christopher Austin MD will serve as the first director of the National Center for Advancing Translational Sciences. 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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Happy Birthday to Baby’s First Test! Genetic Alliance’s Program educate and empower families & health care providers during National Newborn Screening Awareness Month. More > |
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Lysosomal Storage Disorders Survey A student researcher is conducting an interview study of adults with lysosomal storage disorders who have been involved in any clinical study or registry. Access the survey > |
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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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NBS Connect is a new internet-based support network for parents, guardians and individuals with inborn errors of metabolism. Learn more > |
News for Patient Organizations
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A Board Member from a NORD Member Organization Wins Prestigious Award AA&MDSIF Medical Advisory Board member, Dr. Neal Young, has just 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. |
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FDA OOPD Workshop on Pediatric Device Development This free workshop will be held at the FDA's White Oak campus in Silver Spring, Maryland on Monday, September 24. Read more and register here > |
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Gene for Alternating Hemiplegia of Childhood Identified One of NORD's member organizations played a key role in identifying the gene for this disease. Read more > |
News for Medical Professionals
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Proposed Legislation Would Bolster Reporting of Clinical Trial Data The TEST Act would require all US biomedical studies on humans be registered on ClinicalTrials.gov and that the results be reported within one year. More> |
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Conference on Clinical Research for Rare Diseases The NIH Rare Diseases Clinical Research Network and the Clinical and Translational Science Awards are sponsoring the 3rd Conference on Clinical Research for Rare Diseases on October 2. More > |
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CRN Develops Resource for Cystinosis Treatment Cystinosis Research Network develops standards of care resource for families and physicians. Read more > |
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NIH Seeks Proposals to Study Genomic Sequencing in Newborn Period The NIH is intending to fund projects to further the understanding of newborn on-set disorders and to improve treatments for these diseases. Read More > |
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 > |
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Study Compares Oncology Drug Approvals and Costs Tufts study finds oncology drugs approved more quickly in U.S. than in Europe but, in general, more costly. More > |
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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 > |
