Proposed new cut-off levels for values of very long-chain fatty acids, or VLCFAs — whose buildup characterizes X-linked adrenoleukodystrophy (X-ALD) — ... Read more
Treatment with Minoryx Therapeutics’ leriglitazone in a Phase 2/3 clinical trial was found to improve motor function in people with early symptomatic ... Read more
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose ... Read more
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. ... Read more
Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare Artist contest, hosted by the EveryLife ... Read more
The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at ... Read more
Elevated blood levels of a nerve cell-derived component known as neurofilament light chain (NfL) could be a potential biomarker for myelopathy — ... Read more
