Advances in technology and a better understanding of biology have paved the way toward making gene therapy a potential treatment strategy ... Read more
Activation of immune cells resident in the brain is an early event that may contribute to the development of X‐linked adrenoleukodystrophy ... Read more
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 ... Read more
Targeted genetic testing is critical for accurate diagnosis of X-linked adrenoleukodystrophy (ALD), particularly in cases with atypical presentation, the case of ... Read more
[Editor’s Note updating article: According to ALD Life, the PEDALS trial investigating NV1205 as a potential treatment for adrenoleukodystrophy has been placed ... Read more
Minoryx Therapeutics has finished randomizing patients in its ongoing Phase 2/3 clinical trial testing MIN-102, an investigational oral therapy for adrenomyeloneuropathy (AMN) – ... Read more