Written by Valaree DonFrancesco on October 29, 2020
NORD invites you to a virtual externally-led Patient-Focused Drug Development (EL-PFDD) meeting on Krabbe disease, taking place Thursday, October 29, 2020.
If you are a patient, family member or caregiver affected by Krabbe disease, this is an important opportunity to have your voice heard.
EL-PFDD… Read More
Written by Laura Mullen on June 26, 2020
NORD is deeply concerned about the “Promising Pathway Act” recently introduced in both the Senate and the House. Contrary to the stated goal of giving patients with serious and life-threatening diseases a “chance at receiving meaningful treatments,” this legislation, by its own terms, would lower FDA’s approval standards, exposing patients… Read More
Written by Laura Mullen on May 7, 2020
NORD’s recently released COVID-19 Community Survey Report reveals the far-reaching impact the pandemic is having on rare patients and families. 772 participants responded to the survey conducted by NORD’s research team from April 1 through April 8, 2020. Respondents represent 49 of 50 states and Washington,… Read More
Written by Laura Mullen on April 28, 2020
“…As we pull into the hospital, she starts screaming that she doesn’t want to be there, and she wants to be home… No matter how much her doctors want to make her feel better, and she’s also three, so it’s hard to explain to her that this will… Read More
Written by Valaree DonFrancesco on April 23, 2020
The current COVID-19 pandemic necessitates a new approach to many aspects of life—particularly those that involve gathering together. The health and well-being of people living with rare diseases, their loved ones and those working to improve their lives are always top priorities at NORD.
With that in mind, we have made… Read More