New clinical trial for adrenoleukodystrophy, a rare disease of brain metabolism

by Hospital Universitari de Bellvitge

The IDIBELL Neurometabolic Diseases team, which is also part of CIBERER, plans to start the study in early 2022 with the anti-inflammatory dimethyl fumarate in patients with this condition. The study...

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A new model of the worm C. elegans to progress in the study of a rare disease of the nervous system

by Hospital Universitari de Bellvitge

Chromosome X-linked adrenoleukodystrophy (X ALD) is a rare genetic disease in which long-chain fatty acids accumulate in the blood and the nervous tissue, and where the myelin in the neurons is damage...

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Minoryx Therapeutics receives Orphan Drug Designation from the US FDA for its lead candidate MIN-102

by Professional Newco

MIN-102 targets X-linked Adrenoleukodystrophy (X-ALD), a life threatening orphan CNS disease with high unmet medical need. Orphan drug status for MIN-102 has now been granted by both the FDA and the E...

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