News - NORD (National Organization for Rare Disorders)

September 23, 2016

Senate Passes Short-Term Agreement to Accelerate Treatments for 15 Million Sick Children with Rare Diseases

Posted by Jennifer Huron

Unanimous vote extends the Rare Pediatric Disease Priority Review Voucher Program with important policy changes through December

Washington, D.C., September 23, 2016 – On Thursday, the Senate reached an important compromise to accelerate new treatments to help 15 million sick children with rare diseases.

Led by Senators Robert Casey (D-PA), Johnny Isakson (R-GA), and Lamar Alexander (R-TN), the Senate voted unanimously to… Read More

Tags: Advancing Hope Act of 2016 (S.1878), Continuing Resolution, FDA, Peter L. Saltonstall, Rare Pediatric Disease Priority Review Voucher, S.1878, Senator Casey, Senator Isakson, Senator Johnny Isakson (R-GA), Senator Lamar Alexander (R-TN), Senator Robert Casey (D-PA)

September 22, 2016

TOPIC: Advocacy, Press Releases, Research

NORD Converges on Capitol Hill for Rally for Medical Research Hill Day to Advocate for Increased Funding for the National Institutes of Health (NIH)

Posted by Jennifer Huron

More than 300 organizations join forces to ask Congress to provide robust, sustained, and predictable budget increases for the NIH

Washington, D.C., September 22, 2016— The National Organization for Rare Disorders (NORD) is one… Read More

Tags: ally for Medical Research Hill Day, National Institutes of Health (NIH), Paul Melmeyer, Rally for Medical Research, Senate Appropriations Committee, Senator Patty Murray (D-WA), Senator Roy Blunt (R-MO)

September 15, 2016

TOPIC: Advocacy

The Hill publishes NORD op-ed: Congress must act now to help 15 million sick children

Posted by Jennifer Huron

The Hill, a top U.S. political website, read by Congressional offices, the White House, political pundits, and more, today published the following op-ed by NORD President and CEO, Peter L. Saltonstall, urging Congress to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) program.

The PRV program encourages the development… Read More

Tags: Abeona Therapeutics, Advancing Hope Act of 2016, Advancing Hope Act of 2016 (S.1878), advocacy alert, Batten Disease, Congress, duchenne muscular dystrophy, epidermolysis bullosa, George Soros, Krabbe disease, metachromatic leukodystrophy, Nationwide Children’s Hospital, op-ed, Orphan Drug Act, Peter L. Saltonstall, Progeria, PRV Program, Rare Pediatric Disease Priority Review Voucher Program, S.1878, Sanfilippo Syndrome, Sly syndrome, Spinal Muscular Atrophy, Spinocerebellar ataxia autosomal recessive 9 (SCAR9), The Hill, TK2 Mitochondrial DNA Depletion Syndrome

September 14, 2016

TOPIC: Patient Stories, Patients & Members

A Group No One Applies to Join

Posted by Jennifer Huron

By Elizabeth Silva Anderson, ECD Global Alliance, a NORD Member Organization

An interview with Erdheim-Chester Disease patient, Janet Bunge, in honor of Erdheim Chester Disease Awareness Week (#ECDAwareness), Sept. 12-17, 2016

“I belong to ECD. A group which no one applies to join.” The spirited mother of four recounts a tale of searching, frustration, courage, and finally hope.

Janet Bunge was… Read More

Tags: breast cancer, Cancer, Diagnosis, Dr. Eli Diamond, Dr. Juvianee Estrada-Veras, Dr. Rachel Harper, ECD Global Alliance, ECD Patient and Family Gathering, ECD Referral Care Centers, Elizabeth Silva Anderson, Erdheim Chester Disease Awareness Week, Erdheim-Chester Disease, European Union, FDA, histiocytes, Janet Bunge, Lexington Clinic, Membership Organizations, Memorial Sloan-Kettering Cancer Center, MRI, multicentric reticulohistiocytosis, National Cancer Institute, NIH, oncologist, rare cancer

September 13, 2016

TOPIC: Advocacy

ICER Comments Submitted

Posted by Jennifer Huron

The National Organization for Rare Disorders submitted comments to the Institute for Clinical and Economic Review (ICER) in response to their call for comments on how to improve their value framework development process. You can find NORD’s comments here.

People with rare diseases have a uniquely important role to play in value framework development. Almost every input into a value… Read More

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September 23, 2016

Senate Passes Short-Term Agreement to Accelerate Treatments for 15 Million Sick Children with Rare Diseases

Posted by Jennifer Huron

September 22, 2016

NORD Converges on Capitol Hill for Rally for Medical Research Hill Day to Advocate for Increased Funding for the National Institutes of Health (NIH)

Posted by Jennifer Huron

September 15, 2016

The Hill publishes NORD op-ed: Congress must act now to help 15 million sick children

Posted by Jennifer Huron

September 14, 2016

A Group No One Applies to Join

Posted by Jennifer Huron

September 13, 2016

ICER Comments Submitted

Posted by Jennifer Huron

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