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September 1, 2020

TOPIC: Featured News, Medical, Advocacy, Newborn Screening

September is Newborn Screening Awareness Month

Posted at September 9, 2020 08:25 am by Valaree DonFrancesco

A Personal Reflection from Stephanie Cozine, Delaware Rare Action Network Ambassador

Did you know that your baby is screened for different diseases depending on which state you live in?  That it’s possible your state doesn’t screen for a disease that it is recommended to? Do you know what your newborn is screened for and how?

I knew that my baby was… Read More

August 17, 2020

TOPIC: Featured News, Medical, Advocacy

NORD Releases Four Guiding Principles on Telehealth Issues

Posted at August 8, 2020 04:11 pm by Valaree DonFrancesco

The COVID-19 pandemic has had a huge impact on the rare disease community. NORD has heard from countless patients over the last few months about how this crisis has added to the already significant challenges that come from living with, or caring for, someone with a rare disease. Though many challenges remain, the expansion of… Read More

June 22, 2020

TOPIC: Featured News, Medical, Patients & Members

NORD’s New Genome Editing Videos Address Patient/Caregiver Questions

Posted at June 6, 2020 08:31 am by Valaree DonFrancesco


The newest addition to the NORD Rare Disease Video Library is a set of four videos on genome editing, a promising approach to treatment for many people affected by rare diseases. The videos address questions shared with NORD by our members and social media followers.

These short, animated videos answer basic questions such as “What is genome editing?”Read More

April 21, 2020

TOPIC: Featured News, Medical, Research

NORD’s Director of Research Programs Publishes Article on Rare Disease Research in the 2020 Rare Neurological Disease Special Report

Posted at April 4, 2020 03:03 pm by Valaree DonFrancesco

The National Organization for Rare Disorders (NORD®) is excited to spotlight an article written by NORD’s Director of Research, Vanessa Boulanger, MSc, published in the 2020 Rare Neurological Disease Special Report. The feature, “Now Is The Time For Research On Rare Diseases,” details the importance of natural history studies, the role of patients and caregivers as… Read More

April 16, 2020

TOPIC: Featured News, Medical

NORD and FMC’s Gene Therapy Survey: A Call to Action for Healthcare Professional Education

Posted at April 4, 2020 08:46 am by Valaree DonFrancesco

The results of a survey on gene therapy conducted by NORD and Frontline Medical Communications (FMC) among physicians and other healthcare professionals were recently announced. The responses indicated significant knowledge gaps among providers across 14 specialty areas.

For instance:

  • 63% of respondents were unaware of current FDA-approved gene therapies.
  • Respondents reported a low level of comfort in discussing gene therapy… Read More