News

February 25, 2020February 25, 2020

New Worm Model May Help in More Quickly Finding Ways to Treat X-ALD, Study Shows

News

A new genetically modified earthworm model, Caenorhabditis elegans (C. elegans), mimics the features of human X-linked adrenoleukodystrophy (X-ALD), and could help in more quickly finding targets for therapies and at lower cost, a ... Read more

February 20, 2020March 3, 2020

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

News

An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and ... Read more

February 18, 2020February 25, 2020

Researchers Develop Simpler, Faster Method to Screen Newborns for X-ALD

News

Researchers have developed a simpler, faster, reliable method of screening newborns for X-linked adrenoleukodystrophy (X-ALD), which was validated in a North Carolina pilot study. The work suggests that this method could ... Read more

February 11, 2020February 11, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

News

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 6, 2020February 6, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

News

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

February 4, 2020February 4, 2020

Blood Levels of 3 Biomarkers May Help Identify Newborns at Risk for X-ALD, Chinese Study Suggests

News

Measuring the levels of three biomarkers in the blood may help identify newborns at risk of X-linked adrenoleukodystrophy (X-ALD), a Chinese study suggests. The study, “Evaluation of a panel ... Read more

January 28, 2020January 28, 2020

European Medicines Agency Grants Orphan Drug Designation to Dimethyl Fumarate for ALD

News

Dimethyl fumarate — an approved therapy for multiple sclerosis and psoriasis — has been granted orphan drug designation by the European Medicines Agency for the treatment of adrenoleukodystrophy (ALD). Orphan designation is ... Read more

January 21, 2020January 21, 2020

With Attention to Symptoms, Anesthesia Poses Low Risk of Complications for ALD Children, Study Finds

News

If physicians pay close attention to the comorbidities associated with adrenoleukodystrophy (ALD) — particularly gastroesophageal reflux disease (GERD), adrenal insufficiency, and seizures — children with the disease may safely ... Read more

January 14, 2020January 14, 2020

Potential X-ALD Oral Therapy, Leriglitazone, Put on Fast Track by FDA

News

Leriglitazone, a potential therapy for X-linked adrenoleukodystrophy (X-ALD), has been put on a fast track for review and possible approval by the U.S. Food and Drug Administration (FDA). Leriglitazone, ... Read more

January 7, 2020January 7, 2020

Spastic Paraparesia Can Be Only Symptom of Adrenomyeloneuropathy, Case Report Suggests

News

Spastic paraparesia — a condition that involves weakness in the lower limbs and difficulty moving them — can be the only symptom of adult-onset adrenoleukodystrophy (ALD), a case report ... Read more

Pin It on Pinterest