Uncategorized Archives - NORD (National Organization for Rare Disorders)

July 9, 2018

TOPIC: Featured News, Patient Stories, Patients & Members, Uncategorized

Moving documentary on Joshua Frase Foundation, Nibs – the matriarch of the first ever MTM dog colony, and the power of patient advocacy

Posted at July 7, 2018 09:55 am by Laura Mullen

The Joshua Frase Foundation (JFF) is a non-profit organization founded with a simple yet seemingly unattainable vision: to find a cure for Myotubular Myopathy (MTM). The journey towards a cure started the day Joshua Frase was born with a disease so rare that he was said to be only one of fifty five known cases around the… Read More

Tags: Alison Rocket Frase, documentary, Joshua Frase Foundation, MTM, myotubular myopathy, Nibs, Paul Frase

March 2, 2018

TOPIC: Featured News, Patients & Members, Research, Uncategorized

Changing the Landscape of Rare Disease Research

Posted at March 3, 2018 03:09 pm by Laura Mullen

*The following is a a guest blog posting by NORD’s Director of Research Programs, Vanessa Boulanger, originally published 2/28/18 on PCORI.org at https://www.pcori.org/blog/changing-landscape-rare-disease-research?utm_source=general`

A rare disease, by definition, affects fewer than 200,000 individuals in the United States. Up to 30 million Americans have a rare disease, and most conditions are poorly understood. The National Organization for Rare Disorders (NORD), the… Read More

Tags: grants, IAMRARE, NORD, patient-centered, PCORI, Rare disease research

March 27, 2017

TOPIC: Press Releases, Uncategorized

NORD and Neurology Reviews Publish Special Report

Posted at March 3, 2017 03:29 pm by Jennifer Huron

As part of its efforts to educate medical professionals about rare diseases, NORD, the leading independent nonprofit representing the 30 million Americans with rare diseases, is pleased to announce publication of the 3rd annual Rare Neurological Disease Special Report™, with Neurology Reviews®, a Frontline Medical Communications (FMC) print and digital media publication.

Published as a supplement to the… Read More

Tags: grants, Hereditary Neuropathy Foundation, IAMRARE, Keck Graduate Institute, Neurology Reviews, patient perspective, patient-centered, product review, Quincy M.E., Rare Diseases & Orphan Products Breakthrough Summit, Rare Neurological Disease Special Report

September 30, 2016

TOPIC: Uncategorized

President Obama Signs Advancing Hope Act

Posted at September 9, 2016 03:48 pm by Jennifer Huron

Washington, D.C., September 30, 2016 – Today, President Obama signed the Advancing Hope Act (S. 1878), extending the Rare Pediatric Disease Priority Review Voucher (PRV) program until December 31.

“On behalf of the 15 million children with rare diseases, we thank the President for signing the Advancing Hope Act so… Read More

June 28, 2016