February 24, 2021February 24, 2021 New Cut-off Levels for Blood VLCFAs May Aid X-ALD Diagnosis 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 ... Read more
February 17, 2021February 17, 2021 Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients 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 ... Read more
February 17, 2021February 17, 2021 Adrenoleukodystrophy and Diet Adrenoleukodystrophy (ALD) is a rare genetic disease in which very long-chain fatty acids (VLCFAs) build up inside cells in males, causing a number of behavioral ... Read more
February 10, 2021February 11, 2021 Pandemic Won’t Stop Rare Disease Day on Feb. 28 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 ... Read more
February 10, 2021February 10, 2021 Emergency Management of Adrenal Insufficiency in Adrenoleukodystrophy An estimated 80% of men and boys with adrenoleukodystrophy (ALD) develop adrenal insufficiency — when the body does not produce enough of the steroid hormone ... Read more
News, syndicated NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns February 4, 2021February 4, 2021 While progress was made last year on newborn screening and other policy issues critical to ...
News Leriglitazone Improves Motor Function in Early Symptomatic AMN Patients February 3, 2021February 3, 2021 Treatment with Minoryx Therapeutics’ leriglitazone in a Phase 2/3 clinical trial was found to improve motor ...
Health Insight Paramedical Treatment for Adrenoleukodystrophy February 3, 2021February 3, 2021 If you or your child have adrenoleukodystrophy (ALD), you may benefit from paramedical treatments — ...
News, syndicated COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say January 27, 2021January 27, 2021 The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide ...
Health Insight Respite Care for CALD Patients January 27, 2021January 27, 2021 Providing care for a child with cerebral adrenoleukodystrophy (CALD) can leave you physically, mentally, and ...
News, syndicated NORD’s Caregiver Respite Program Continues Through Pandemic January 20, 2021January 20, 2021 Caring for a loved one with a rare disease, especially during these uncertain times, demands ...
Art Contest Salutes Creativity of Rare Disease Communities January 19, 2021January 19, 2021 Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare ...
Ways Extended Family Can Help in Care for Child With CALD January 19, 2021January 20, 2021 If there’s a child in your extended family with childhood cerebral adrenoleukodystrophy (CALD), you know how ...
NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum January 13, 2021January 13, 2021
Blood Neurofilament Light Chain Levels May Be Myelopathy Biomarker in ALD January 7, 2021January 7, 2021
EURORDIS Award Contest Welcomes Photos of Life With Rare Disease December 18, 2020December 18, 2020 The annual EURORDIS Photo Award contest is inviting people worldwide to visually express what life ...
NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting December 3, 2020December 3, 2020
Autobahn Plans to Start Clinical Development of AMN Candidate ABX-002 in 2021 December 2, 2020December 2, 2020 Autobahn Therapeutics has successfully completed a pre-investigational new drug (IND) meeting with the U.S. Food ...
Individualized Education Plan (IEP) for Children With Adrenoleukodystrophy December 2, 2020December 2, 2020
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe November 26, 2020November 26, 2020
News EUnetHTA Seeks Patient, Caregiver Input for Joint Assessment on CALD Therapy November 25, 2020November 25, 2020 EUnetHTA, Europe’s health technology assessment network, is seeking input from children and adolescents, ages 18 ...
Health Insight Tips for Dealing With Fatigue When You Have ALD November 25, 2020November 25, 2020 Fatigue is a common symptom for patients with chronic diseases such as adrenoleukodystrophy (ALD). Fatigue ...
News, syndicated Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry November 19, 2020November 19, 2020 Leaders in the U.S. rare disease community came together recently for a webinar to present ...
News High Biotin Doses Restore Nerve Cell Health in ALD Mice, Study Shows November 18, 2020November 18, 2020 High doses of the B vitamin biotin restored metabolic balance and rescued nerve degradation in ...
News Trials Testing Leriglitazone Get $29.4M in Funding From European Investment Bank November 11, 2020November 11, 2020 Clinical trials testing Minoryx Therapeutics‘ leriglitazone (MIN-102), an investigational oral therapy for all forms of ...
Health Insight Medications With No Clear Benefit for Adrenoleukodystrophy November 11, 2020November 11, 2020 A number of medications have been proposed as treatment options for adrenoleukodystrophy (ALD) and have ...
News, syndicated NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits November 4, 2020November 4, 2020 The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of ...
Health Insight Treatment Plan for Adrenoleukodystrophy November 4, 2020November 4, 2020 Adrenoleukodystrophy is a rare genetic brain disorder that has been categorized into three different forms: ...
News, syndicated UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner October 28, 2020October 28, 2020 A photo of a bespectacled young boy, his red baseball cap slightly askew as he ...
