Posts by: Alice Melao

News

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

July 30, 2019July 30, 2019

Report Describes New ABCD1 Variant Linked to X-ALD in Chinese Boy

News

A new genetic mutation in the ABCD1 gene was identified and linked to X-linked adrenoleukodystrophy (X-ALD) in a young Chinese boy, a new case report revealed. The case was described in ... Read more

April 23, 2019April 23, 2019

Brain Resident Immune Cells May Play Role in X-ALD Progression, Study Suggests

News

Activation of immune cells resident in the brain is an early event that may contribute to the development of X‐linked adrenoleukodystrophy (X-ALD) and progressive white matter loss, a study suggests. ... Read more

April 10, 2019April 10, 2019

Cholesterol-derived Molecule 25-HC May Hold Potential for Severe Type of X-ALD

News

Treatment with a naturally occurring cholesterol-derived compound, called 25-hydroxycholesterol (25-HC), may represent a new strategy to prevent the accumulation of long-chain fatty molecules in X-linked adrenoleukodystrophy (X-ALD), results from a ... Read more

January 29, 2019January 29, 2019

Targeted Genetic Testing Is Critical for X-Linked ALD Diagnosis in Atypical Cases, Report Shows

News

Targeted genetic testing is critical for accurate diagnosis of X-linked adrenoleukodystrophy (ALD), particularly in cases with atypical presentation, the case of an adult male with mild symptoms demonstrates. The case, ... Read more

December 21, 2018January 9, 2019

NeuroVia Starts Phase 1/2 Trial to Evaluate NV1205 in Childhood Cerebral Adrenoleukodystrophy

News

[Editor’s Note updating article: According to ALD Life, the PEDALS trial investigating NV1205 as a potential treatment for adrenoleukodystrophy has been placed on a temporary hold by NeuroVia. A reason for ... Read more

December 19, 2018December 19, 2018

Phase 2/3 Trial of MIN-102 as Potential Treatment for AMN Ready to Dose Patients, Minoryx Reports

News

Minoryx Therapeutics has finished randomizing patients in its ongoing Phase 2/3 clinical trial testing MIN-102, an investigational oral therapy for adrenomyeloneuropathy (AMN) – the most common type of X-linked adrenoleukodystrophy (X-ALD). A total ... Read more