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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 ... 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 ... 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 ... 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 ... 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. ... 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 ... Read more

November 5, 2019December 3, 2019

Aging Raises Risk of Women Developing ALD Symptoms or More Severe Disease, Study Suggests

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Age is the major risk factor for adrenoleukodystrophy (ALD) symptoms and their severity in women carrying one mutated copy of the ABCD1 gene — the gene associated with ALD, an Italian study suggests. ... Read more

October 29, 2019October 29, 2019

Researchers Develop Efficient Method to Screen for ALD in India, Study Reports

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A rapid and efficient method to screen newborns or people at risk for adrenoleukodystrophy (ALD) in India has been developed, according to a new study. The study, “Flow injection ... Read more

October 22, 2019October 22, 2019

Immune Cells in Blood Samples from X-ALD Patients May Predict Response to Treatment, Study Says

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Immune cells derived from blood samples of people with adrenoleukodystrophy (X-ALD) may help in evaluating their inflammatory status of patients and likely response to treatment, an early study suggests. The ... Read more

October 15, 2019October 15, 2019

Most ALD Patients Without MRI Lesions Retain Normal Cognitive Function, Study Suggests

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Most men with adrenoleukodystrophy (ALD) who show either minimal or no visible lesions in magnetic resonance imaging (MRI) scans appear to retain normal cognitive abilities, a study has found. ... Read more

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