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January 14, 2020January 14, 2020

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

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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, also ... Read more

January 7, 2020January 7, 2020

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

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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 suggests. ... Read more

December 17, 2019December 17, 2019

Consider AMN Diagnosis in Men with Spastic Paraplegia Symptoms, Chinese Study Suggests

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Diagnosis of adrenomyeloneuropathy (AMN) — the adult-onset form of adrenoleukodystrophy (ALD) — should be considered in men with symptoms suggestive of hereditary spastic paraplegia, such as weakness in the legs, a Chinese study ... Read more

December 10, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. ... Read more

December 5, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking ... Read more

December 3, 2019December 3, 2019

Retina Thickness Linked to Neurodegeneration in Spinal Cord of ALD Patients, Study Finds

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Thinning of the layer of nerve cells that form the back of the eye, or retina, is linked to neuron degeneration in the spinal cord of people with adrenoleukodystrophy (ALD), ... Read more

November 26, 2019November 26, 2019

Three New Mutations Associated With ALD Identified in Iranian Study

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Researchers found three new ABCD1 mutations associated with adrenoleukodystrophy (ALD) in three Iranian families. The cases were reported in “Novel ABCD1 gene mutations in Iranian pedigrees with X-linked adrenoleukodystrophy,” a study ... Read more

November 19, 2019November 19, 2019

Diffusion MRI Shows Potential as Sensitive Measure of ALD Progression, Study Shows

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Diffusion magnetic resonance imaging — which measures the random motion of water molecules in tissues — detected neurological degeneration in people with adrenoleukodystrophy (ALD), even when they did not show ... Read more

November 14, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: ... Read more

November 12, 2019November 12, 2019

Cell-based Gene Therapy Represents ‘Hope’ for X-ALD Patients, Review Study Suggests

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Advances in technology and a better understanding of biology have paved the way toward making gene therapy a potential treatment strategy for rare genetic disorders, including neurometabolic diseases like X‐linked ... Read more