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Adrenoleukodystrophy; Other names: X-linked adrenoleukodystrophy, ALD, X-ALD, Siemerling–Creutzfeldt disease, bronze Schilder disease: White matter, with reduced volume and increased signal intensity. The anterior white matter is spared. Features are consistent with X-linked adrenoleukodystrophy. Pronunciation /-
Elivaldogene autotemcel, sold under the brand name Skysona, is a gene therapy used to treat cerebral adrenoleukodystrophy (CALD). It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in May 2018.
Leriglitazone was intended for the treatment of cerebral adrenoleukodystrophy. [5] References
Elivaldogene autotemcel (Skysona): treatment for cerebral adrenoleukodystrophy [9] Etranacogene dezaparvovec (Hemgenix): AAV-based treatment for hemophilia B [10] Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [11] Gendicine: treatment for head and neck squamous cell carcinoma
ALBUQUERQUE, N.M. (KRQE) – It’s a rare genetic disorder that impacts a small population, and due to limited research, much remains unknown about treating adrenoleukodystrophy. Now, there’s a ...
The one exception to this is any type of leukodystrophy carried on a sex chromosome, such as X-linked adrenoleukodystrophy, which is carried on the X-chromosome. Because of the inheritance pattern of X-linked diseases, males are more often affected by this type of leukodystrophy, while female carriers are often symptomatic, though not as ...
PXL065 (formerly known as DRX-065) has demonstrated preclinical efficacy for both NASH [1] and X-linked adrenoleukodystrophy (X-ALD). [2] In 2022, it successfully completed a 9 month Phase 2 trial in biopsy-proven NASH patients where it met the primary endpoint for reduction in liver fat without weight gain or edema. [3] [4] [5]
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