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November 5, 2019November 5, 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

October 8, 2019October 8, 2019

Researchers Discover New Genetic Mutation Among 6 Chinese Men with AMN

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A new study in six Chinese men with adrenomyeloneuropathy (AMN) revealed a novel ABCD1 gene mutation, alterations such as skin hyperpigmentation, spasticity, and sexual dysfunction, as well as brain lesions ... Read more

October 1, 2019October 1, 2019

Symptoms of Peroxisomal Disorders Like X-ALD Often Mistaken for Another Disease, Study Finds

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Peroxisomal disorders, including adrenoleukodystrophy, are evident in a relatively high number of Chinese patients whose symptoms are similar to those caused by hereditary spastic paraplegia (HSP), highlighting the importance ... Read more

September 27, 2019September 27, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

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Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more

September 24, 2019September 24, 2019

Magenta Therapeutics’ Stem Cell Therapy MGTA-456 Earns FDA’s Regenerative Medicine Advanced Therapy Designation

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The U.S. Food and Drug Administration (FDA) has granted Magenta Therapeutics’ cell therapy MGTA-456 the Regenerative Medicine Advanced Therapy (RMAT) designation for treating inherited metabolic disorders, including cerebral adrenoleukodystrophy. The RMAT ... Read more

September 19, 2019September 19, 2019

Clinical Trial Data Support Safety, Efficacy of Lenti-D for Boys with CALD

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New Phase 2/3 clinical trial data suggests that the gene therapy candidate Lenti-D is safe and effective for the treatment of cerebral adrenoleukodystrophy (CALD), Bluebird Bio announced. The ... Read more

September 3, 2019September 3, 2019

Lack of SIRT1 Likely Contributes to Neuroinflammation in X-ALD, Study Says

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Lack of a protein called sirtuin 1 (SIRT1) in cells that build up the lining of small blood vessels in the brain likely contributes to neuroinflammation associated with ... Read more

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