Welcome to NORD

NORD President Introduces FDA Webinar Series

NORD President and CEO Peter L. Saltonstall introduces a series of educational webinars developed by FDA and NORD on the drug approval and development process. More >

Join NORD in Opposing Repeal of Orphan Drug Tax Credit (ODTC)

Rare disease patient organizations can sign-on to NORD's letter to Congress on this issue. Organizations can get involved here and individuals can send a message here.

U.S. Senate Approves Rare Disease Day Resolution

NORD thanks Senators Brown (D-OH) & Barrasso (R-WY) for introducing a resolution in support of Rare Disease Day that the Senate approved. More >

National PKU Alliance Creates New Video

A video produced in partnership with the National PKU Alliance illustrates the daily challenges of living with PKU and why staying on the diet for life is so essential.  More >

PCIP Will Extend Coverage Through March

The federally-run PCIP will offer the option of two additional months of coverage to people currently enrolled in PCIP who have not yet found new health insurance coverage. More >

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 >

UAB Launches Undiagnosed Diseases Program

The University of Alabama at Birmingham (UAB) has created a new Undiagnosed Diseases Program for cases where a diagnosis has not been made despite extensive efforts by the referring physician. More >

Million Dollar Bike Ride for Rare Disease Research

The 1st Million Dollar Bike Ride to raise money for rare disease research, organized by the Penn Center for Orphan Disease Research and Therapy and Rare Disease Cycling, will be held May 3. More >

New Video about Pushing MPN Research Forward

The story of a group of patients who have invigorated research into a group of blood cancers collectively known as myeloproliferative neoplasms. More >

The Alpha-1 Foundation and Alpha-1 Association Send Letter

The Alpha-1 Foundation and Alpha-1 Association have written a letter to health insurer Humana urging the company to reverse its decision on coverage. More >

FDA Launches Nomination Portal

FDA has launched an advisory committee membership nomination portal that allows interested individuals to submit online nominations for advisory committees. More >

New NCHPEG Programs for Pediatricians and Neurologists

NCHPEG, The Jackson Lab, and partners have announced the launch of 2 new programs for pediatric and neurology providers. More here and here.

Diagnosis and Treatment Recommendations for NMO

The German Neuromyelitis Optica (NMO) Study Group has published a report stating that testing for the serum marker AQP4-Ab and imaging techniques are necessary for the diagnosis of NMO. More>

ACMG Guidelines for Management of PKU

The American College of Medical Genetics has published a new guideline that supports the need for life-long management of phenylalanine levels in individuals with PKU. More >

Rare Coagulation Disorders Resource Room

The Resource Room provides information for very rare and heterogeneous coagulation disorders.  More >

OOPD Announces Pediatric Device Consortia Grant Recipients

Through its Pediatric Device Consortia Grant Program, FDA has awarded 7 grants to advance the development of pediatric medical devices. More >

Database Matches Cancer Genes to Drugs

The Drug-Gene Interaction database is for individuals who want to know whether specific gene mutations in cancer patients could be targeted with existing drug therapies. More >

UK Rare Disease Strategy Launched

The UK government has launched a strategy to improve diagnosis, research, and treatment of rare diseases. More >

Article from NORD Staff Member in The Source

NORD's Sr. VP, Medical & Scientific Affairs, J Russell Teagarden recently wrote an article entitled "Patient Centeredness in Health Research: The Challenge in Rare Diseases". More >