Welcome to NORD
News and Events
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NORD Research Grants Awarded for 2013 RFPs NORD’s Medical Advisory Committee has recommended, and the NORD Board of Directors has approved, the awarding of nine research grants. More > |
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119 Patient Organizations Stand Together in Support of ODTC Read NORD's letter signed by 119 patient organizations urging Congress not to repeal the Orphan Drug Tax Credit. More > Send your own letter > |
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View Video of NORD's Capitol Hill Briefing On March 20, NORD hosted a briefing on patient access to treatments and related topics. A video has now been posted. More> |
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A recent study published in JAMA has led to debate about whether FDA should employ a "one size fits all" approach to drug reviews. Read NORD's position on this. |
News for Patients
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National Hemophilia Foundation Meeting Nominations are being sought for the 2014 Annual Meeting Awards of Distinction in Programs and Awards of Excellence. The deadline for submissions is May 19. More > |
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The NephCure Foundation has launched the NephCure Kidney Network, a registry of people with rare diseases that cause primary nephrotic syndrome. More > |
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Online Communication Guide for Clinical Trial Participants CISCRP and Langland have launched “Speak Out, But Speak Smart” to encourage discussion about clinical trial participation. More > |
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An Assessment of Parental Coping Strategies and Positive Growth This survey is being conducted by the University of Newcastle, Australia, parents and caregivers of a child with a diagnosed or undiagnosed neurogenetic disorder. More > |
News for Patient Organizations
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FDA has a calendar on its website with news about upcoming public meetings. More > |
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New Video from the Myotonic Dystrophy Foundation This video features patients, caregivers and researchers as they describe living with myotonic dystrophy and the current research advances. More > |
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Patient Advocacy Leadership (PAL) Awards Genzyme has launched its 4th annual PAL Awards program for organizations that serve the lysosomal storage disorder patient community. Proposals must be received by June 6. More > |
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Public Meeting on PAH Patient-Focused Drug Development FDA will host a public meeting on patient-focused drug development for pulmonary arterial hypertension (PAH) on May 13. More > |
News for Medical Professionals
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PhenomeCentral is a secure data repository for clinicians and scientists working in the rare disease community to share information. More > |
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ACMG Guidelines for Genetic Testing for Inherited Colorectal Cancer ACMG has published standards and guidelines for genetic testing for Lynch syndrome, familial adenomatous polyposis, and MYH-associatedpolyposis. More> |
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EnRich Innovative Trial Design Course This course is designed for pediatric healthcare and research professionals with experience in clinical trial development and an interest in better designed trials. More > |
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Overcoming Challenges in Clinical Trials of Rare Genetic Diseases Rare disease patients and researchers are eager to connect to advance clinical research, but there are many obstacles, this report offers some insight from a PKU trial. More> |
News for Industry
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President Signs Gabriella Miller #KidsFirst Research Act Into Law This law will redirect wasteful spending on political conventions to important pediatric research. More > |
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Fiscal Year 2015 FDA Proposed Budget Announced The President is requesting a $4.7 billion budget for FDA, which is an 8.1% increase over the 2014 budget Congress passed earlier this year. More > |
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FDA Issues First Pediatric Rare Disease Priority Review Voucher FDA issued the first Priority Review Voucher for a pediatric rare disease application, as authorized in the FDA Safety and Innovation Act of 2012. More> |
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27 New Novel Drugs Approved in 2013 The FDA’s Center for Drug Evaluation and Research approved 27 new molecular entities in 2013, and one-third of these were approved to treat rare disorders. More > |
