News

February 24, 2021August 23, 2022

New Cut-off Levels for Blood VLCFAs May Aid X-ALD Diagnosis

News

Proposed new cut-off levels for values of very long-chain fatty acids, or VLCFAs — whose buildup characterizes X-linked adrenoleukodystrophy (X-ALD) — show higher sensitivity and specificity for detecting genetically confirmed ... Read more

February 17, 2021February 17, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

News, syndicated

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 10, 2021February 11, 2021

Pandemic Won’t Stop Rare Disease Day on Feb. 28

News, syndicated

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the ... Read more

February 4, 2021February 4, 2021

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

News, syndicated

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs ... Read more

February 3, 2021February 3, 2021

Leriglitazone Improves Motor Function in Early Symptomatic AMN Patients

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Treatment with Minoryx Therapeutics’ leriglitazone in a Phase 2/3 clinical trial was found to improve motor function in people with early symptomatic adrenomyeloneuropathy (AMN), one of the most common types of ... Read more

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 to ... 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 for ... 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 rare ... 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 Patient ... 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 (ALD), ... Read more