Rare Disease News

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

Published November 13, 2020 by NORD

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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 …

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

Published November 3, 2020 by NORD

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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 …

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

Published November 2, 2020 by NORD

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[carousel id=”48517″] 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 components. FDA regulators, academic …

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

Published October 28, 2020 by NORD

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Washington, DC, October 28, 2020—As 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 …

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RDCA-DAP First Year Milestones Highlighted at Virtual Workshop

Published October 21, 2020 by NORD

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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 …

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NORD Issues Report on Ensuring Access to Telehealth for Rare Diseases

Published October 15, 2020 by NORD

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For those in the rare community, one of the few positives that have come from the global COVID-19 pandemic has been the wider availability of telehealth services. Through surveys, virtual …

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NORD Highlights New Era of Innovation and Public Health Awareness in Virtual 2020 Rare Diseases and Orphan Products Breakthrough Summit

Published October 9, 2020 by NORD

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Washington, DC, October 9, 2020—On October 8 and 9, the National Organization for Rare Disorders (NORD®) virtually hosted the 2020 Rare Diseases and Orphan Products Breakthrough Summit, the first time the organization representing over 25 million Americans impacted …

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NORD and FDA Co-Host Public Listening Session, Focusing on COVID-19 and Rare Diseases

Published October 5, 2020 by NORD

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The COVID-19 pandemic has caused great fears around drug shortages, the demand for personal protective equipment (PPE) and the status of clinical trial research for rare diseases. On September 16, …

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Nebraska Medicaid Expansion Is Progress for Rare Disease Patients, Highlights Critical Need to Support Medicaid During COVID-19

Published October 1, 2020 by NORD

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Today, Nebraska’s Medicaid expansion begins to take effect. This milestone—two years in the making—means that thousands more low-income people, including many who have a rare disease, now have access to …

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NORD Launches Innovative Natural History Study on Metachromatic Leukodystrophy (MLD)

Published October 1, 2020 by NORD

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Washington, DC, 10/1/2020 — Today, the National Organization for Rare Disorders (NORD®) opened registration for a natural history study on metachromatic leukodystrophy (MLD), a lethal rare disease currently without an FDA–approved treatment. NORD’s Natural History Of MEtachromatic Leukodystrophy (HOME) Study represents an opportunity to address an …

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