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

Quentin M. Anstee, B.Sc., M.B.B.S., Ph.D., M.R.C.P.; Ann K. Daly, B.Sc., Ph.D.; Christopher P. Day, B.A.,M.B., B.Chir.,M.A. (Cambridge), Ph.D.,M.D., F.R.C.P., F.Med ...

It is either tragically funny or devastatingly sad that while a genetic counselor explained the random lunacy of my son’s ...

It’s a rare genetic disorder that impacts a small population, and due to limited research, much remains unknown about ...

X-Linked Adrenoleukodystrophy (X-ALD ... A public health program that tests newborns for certain genetic, endocrine, and metabolic disorders shortly after birth to enable early diagnosis and ...

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 ...

And while Wood said the pharma still isn’t interested in joining the overcrowded obesity landscape, GSK is considering ways to build past the GLP-1 space.

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.