Given the rise in newborn screening for ALD since 2019, and the untapped potential of existing genetic research, the Halls ...

X-linked adrenoleukodystrophy (X-ALD) is a debilitating disease caused by mutations in the ABCD1 gene. Approximately 30% of affected boys develop the progressive ...

Like the majority of clinically important diseases, ALD and NAFLD are complex disease traits where genetic factors (summarized in Table 3 and Table 4) and environmental influences combine to ...

The prevalence of ALD is estimated as between one in 20,000 and one in 50,000 individuals worldwide, and AMN accounts for around 25% to 46% of all ALD cases. Gene therapies have already been ...

Inspired by their 9-month old son William, who was diagnosed with ALD after a newborn screening, the Hall family seeks to jumpstart research into the rare disease, in partnership with The Dallas ...

The patients received the one-time autologous hematopoietic stem cell-based gene therapy as a treatment for early cerebral ...

One of the unexplored frontiers in ALD is the enhancement of ALD reactions using non-thermal methods. We are exploring the ability of low energy electrons to enhance ALD reactions. The goal is to ...

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.