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November 13, 2020

TOPIC: Featured News, Patients & Members, Advocacy, Head of the Herd

Head of the Herd: Charlene Son Rigby, President and Cofounder, STXBP1 Foundation

Posted at November 11, 2020 08:51 am by Valaree DonFrancesco

At the Head of the Herd is… Charlene Son Rigby, President and Cofounder, STXBP1 Foundation

Charlene advocates for those affected by… STXBP1 encephalopathy

Charlene is located in… San Francisco, CA

Charlene most frequently checks her… Twitter account @charleneson

How Charlene got here…  My daughter, Juno, was diagnosed with STXBP1 encephalopathy in 2016 after a three-year journey to find an explanation for her condition…. Read More

November 3, 2020

TOPIC: Featured News, Patients & Members, Advocacy

NORD and Coalition Representing Millions of Patients Urge Policymakers to Adopt “Patient-First” Policy Agenda

Posted at November 11, 2020 08:12 am by Heidi Ross

NORD joined a coalition of 33 other patient organizations representing millions of people with pre-existing conditions to release our top priorities for a new administration. “The 100 Days Agenda: A Patient-First Blueprint” is a first-of-its-kind collaboration that outlines specific steps the president and other elected officials can immediately take to protect patients in the first 100 days… Read More

November 2, 2020

TOPIC: Featured News, Patients & Members, Research, Advocacy, Industry

NORD and Patient Advocacy Groups Host an EL-PFDD Meeting on Krabbe Disease

Posted at November 11, 2020 02:25 pm by Debbie Drell

One of the most important gatherings of the Krabbe disease community occurred in collaboration with the US Food and Drug Administration (FDA) on October 29, and it was 100% virtual with live, interactive componentsFDA regulators, academic researchers and industry drug developers came together with Krabbe disease patients, caregiversRead More

October 28, 2020

TOPIC: Press Releases, Featured News, Patients & Members, Research, Advocacy, RareLaunch

NORD Announces RareLaunch Workshops to Support Those Seeking to Start Rare Disease Nonprofits or Research Programs 

Posted at October 10, 2020 09:37 am by Valaree DonFrancesco

Washington, DC, October 28, 2020As part of its commitment to community engagement and organizational capacity building, the National Organization for Rare Disorders (NORD®) RareLaunch® training program will host two days of free workshops to take place in December, funded in part through a grant from the… Read More

October 21, 2020

TOPIC: Press Releases, Featured News, Patients & Members, Research, Advocacy, Industry, RDCA-DAP

RDCA-DAP First Year Milestones Highlighted at Virtual Workshop

Posted at October 10, 2020 11:14 am by Valaree DonFrancesco

TUCSON, Ariz. and WASHINGTON, D.C. October 21, 2020 — The Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD®) hosted the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) 2020 Virtual Workshop on Monday, October 19. The platform, funded by a cooperative agreement through the U.S. Food and Drug Administration (FDA), serves as… Read More