Rare Disease News

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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Voices of Rare Cancer: Courtney’s Story

Published September 30, 2020 by NORD

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#RareCancerDay brings attention to the fact that without research funding, the only option for patients is to endure blanket therapy regimes that are designed for other types of cancers and, …

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TODAY IS RARE CANCER DAY! Voices of Rare Cancer: Lisa’s Story

Published September 30, 2020 by NORD

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In this week’s Voices of Rare Cancer feature, Lisa shares her experience with cholangiocarcinoma.

Newborn Screening: The HCP Perspective on the Importance of Confirmatory Testing and Collaboration

Published September 29, 2020 by NORD

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SCREENING NEWBORNS FOR RARE DISEASES: THE IMPORTANCE OF CONFIRMATORY TESTING AND COLLABORATION By José Abdenur, MD and Rebekah Bressi, CGC ”Newborn screening is one of the most successful public health …

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A Cure In Sight, in partnership with the UCSF Ocular Oncology Center and NORD, Launches Landmark Ocular Melanoma Natural History Study

Published September 28, 2020 by NORD

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Cleveland, OH, September 28, 2020—A Cure in Sight and the National Organization for Rare Disorders have launched an innovative study to research ocular melanoma, a disease that can cause blindness, …

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Voices of Rare Cancer: Jan’s Story

Published September 25, 2020 by NORD

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My name is Jan and in January 2018 life changed for my husband and me as we knew it. I was on the operating room table to have gastric sleeve surgery. I had six months …

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NORD Joins Coalition Representing Millions of Patients to Release Telehealth Principles

Published September 24, 2020 by NORD

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For Immediate Release Major Groups Representing Millions of Patients Release Telehealth Principles September 24, 2020 – A coalition of organizations representing millions of patients who live with serious, acute, and chronic …

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Head of the Herd: Susan Thornton, CEO, Cutaneous Lymphoma Foundation

Published September 24, 2020 by NORD

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At the Head of the Herd is… Susan Thornton, CEO, Cutaneous Lymphoma Foundation Susan advocates for those living with… Cutaneous lymphoma Susan is located in… Philadelphia, PA. You can connect …

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