We have incredible news to share! Massachusetts-based bluebird bio, a leader in the research and treatment of cancer and severe genetic diseases, just received FDA “Breakthrough Therapy” designation for their Lenti-D™ gene therapy for patients with cerebral adrenoleukodystrophy (CALD). This designation will allow for expedited development and review of the treatment protocol, meaning it is …Read More
Brian’s Hope is proud to share that bluebird bio in Cambridge, MA has announced that interim data for the treatment of cerebral adrenoleukodystrophy (CALD) will be presented during the Clinical Trials at the American Academy of Neurology (AAN) 2016 Annual Meeting on April 20, 2016. Read the complete press release!
It is bittersweet but good to know the process for ALD newborn screening is working in CT. In January, our first CT baby to have ALD was identified. The child is in the care of specialists and will receive the appropriate monitoring and treatments, which if given in the early phase, dramatically improve the outcome …Read More
On February 17th, 2016, the Secretary of the Department of Health & Human Services, Silvia Burwell, approved the addition of adrenoleukodystrophy (ALD) to the Recommended Uniform Screening Panel (RUSP), the federal list of genetic diseases recommended for state newborn screening programs. The addition of ALD to the RUSP will expedite the process of adding ALD …Read More
We are grateful to FOXCT’s Sarah Cody’s follow-up piece on newborn screening for ALD in CT. Hoping CT will be ready to test in a few months. If you don’t look for it you won’t find it until it’s too late. We advocates will be working on this until all states are testing. It shouldn’t …Read More