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.

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 ...

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 ...

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

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 ...

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 ...

For several decades, the prevalence of steatotic liver disease (SLD) has been steadily increasing.1 Alterations in hepatic ...

The "Adrenoleukodystrophy Treatment Market"is anticipated to increase from USD xxx billion in 2023 to USD xxx billion by 2031, with a strong compound annual growth rate (CAGR) of xxx% from 2024 to ...