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September 13, 2021

TOPIC: Press Releases, Advocacy

NORD Statement on Proposed Changes to the Orphan Drug Tax Credit

Posted by Rohan Narayanan

The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the more than 25 million Americans with rare diseases, issued the following statement in response to upcoming congressional consideration of the Build Back Better Act in the House Committee on Ways and Means:

Peter Saltonstall, CEO and President of NORD, stated: “This week, the Ways and Means… Read More

September 10, 2021

TOPIC: Patients & Members, Patient Stories, Voices of Rare Cancer

Sharing Makayla’s Voice for Rare Cancer Day

Posted by Rohan Narayanan

At the age of 14, I was diagnosed with a rare form of GI tract cancer called gastrointestinal stromal tumors (GIST). 

Living with a rare disease is incredibly complicated, and you must learn how to be your own best advocate. Someone else may have the same disease, but everyone’s experience is going to be drastically different. 

Initially, there was not a lot of knowledge of GIST. Although… Read More

September 3, 2021

TOPIC: Patients & Members, Patient Stories, COVID-19, Voices of Rare Cancer

Martin’s Fight Against Waldenstrom Macroglobulnemia

Posted by Rohan Narayanan

I have been a healthy person my whole life. I never drank alcohol or smoked, and I had a good diet. In 2004, I began to have some questionable numbers on blood work, but my primary doctor could never really tell what was going on. My knees shook when I was referred to a hematologist and oncologist in 2010. I was given a diagnosis of Monoclonal gammopathy of undetermined significance (MGUS). I was told that I had an indolent lymphoma that may or may not develop, and that I should “go… Read More

September 1, 2021

TOPIC: Featured News, Newborn Screening

Newborn Screening: History, Future and Awareness Month

Posted by Valaree DonFrancesco

Every September, we celebrate and raise awareness for newborn screening (NBS) in the United States and the impact it has had on millions of families. Each year, approximately four million babies are screened for serious disorders that are present at birth. Of those four million, screening identifies over 12,000 infants annually with a condition that, if left undiagnosed and untreated, would cause… Read More

September 1, 2021

TOPIC: Press Releases

Breaking Down Data Siloes: New Analytics Platform Sparks Opportunity for Rare Disease Field

Posted by Valaree DonFrancesco

TUCSON, Ariz. and WASHINGTON, September 1, 2021 — For two years, Critical Path Institute (C-Path), the National Organization for Rare Disorders (NORD) and the U.S. Food and Drug Administration (FDA) have joined with others throughout the rare disease community to create a novel, best-in-class platform to accelerate rare disease treatment innovation.

On Tuesday, September 14, 10 a.m. – 3:45 p.m. ET,Read More