News

January 27, 2021January 27, 2021

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

News, syndicated

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including ... Read more

January 20, 2021January 20, 2021

NORD’s Caregiver Respite Program Continues Through Pandemic

News, syndicated

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization ... Read more

January 19, 2021January 19, 2021

Art Contest Salutes Creativity of Rare Disease Communities

News, syndicated

Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare Artist contest, hosted by the EveryLife Foundation for Rare Diseases to heighten awareness about ... Read more

January 13, 2021January 13, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

News, syndicated

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 7, 2021January 7, 2021

Blood Neurofilament Light Chain Levels May Be Myelopathy Biomarker in ALD

News

Elevated blood levels of a nerve cell-derived component known as neurofilament light chain (NfL) could be a potential biomarker for myelopathy — spinal cord degeneration — in patients with adrenoleukodystrophy ... Read more

December 18, 2020December 18, 2020

EURORDIS Award Contest Welcomes Photos of Life With Rare Disease

News

The annual EURORDIS Photo Award contest is inviting people worldwide to visually express what life is like with a rare disease, while raising awareness through their work of these ... Read more

December 3, 2020December 3, 2020

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders ... Read more

December 2, 2020December 2, 2020

Autobahn Plans to Start Clinical Development of AMN Candidate ABX-002 in 2021

News

Autobahn Therapeutics has successfully completed a pre-investigational new drug (IND) meeting with the U.S. Food and Drug Administration (FDA) regarding the development plan for its therapy ABX-002, a thyroid ... Read more

November 26, 2020November 26, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to ... Read more

November 25, 2020November 25, 2020

EUnetHTA Seeks Patient, Caregiver Input for Joint Assessment on CALD Therapy

News

EUnetHTA, Europe’s health technology assessment network, is seeking input from children and adolescents, ages 18 and younger, with early cerebral adrenoleukodystrophy (CALD), for a new Joint Assessment on a ... Read more