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Click below to see where
I’ve been so far!
(It’s mind boggling!)
This past year has brought about some exciting changes for the direction Fight ALD will be taking now that I have completed my ALD Awareness Across America mission. To begin with, I was asked if I would be interested in assisting in forming an alliance with other organizations from around the world, which of course, I jumped on board immediately. We initially brought 6 organizations together and formed the ALD-AMN Global Alliance which now consists of 12 and is still growing. It includes charity’s from France, Australia, Germany,
Canada, the UK and many throughout the US. We have so far advanced the submission for newborn screening at the
federal level and are awaiting approval so we can institute it state by state.
New York passed legislation last year and has since identified 12 newborns with the gene since the first of the year. California signed a bill into law last September but will not begin testing until we get HRSA to add it to their recommended panel of diseases.
We also pooled resources to fund a clinical study at the University of Minnesota and have joined a coalition of physicians who specialize in treating patients with ALD and AMN who have begun ALD Connect. You can learn more about their work at
I can’t begin to tell you how excited I am to be involved in all of this and at the prospect of seeing my goal of ALD Awareness along with a cure, come to fruition.
Bluebird bio has begun a clinical study for gene therapy and is taking applications. They hope to enroll 15 prospective patients and have begun treatment at the Boston Children’s Hospital with other locations to follow. All expenses, including travel for the families, will be covered. You can learn more about the study at and
Just to give a quick overview, gene therapy is a less radical procedure to bone marrow transplant (BMT) as it uses the boys own stem cells and inserts 2 copies of the missing ABCD1 gene that will hopefully enable the boys to make the missing protein that breaks down the VLFCA’s. Boys to be considered for the study must be 17 or younger with a Loews score between 0.5 - 9 (inclusive) on the 34 point scale and ii gadolinium enhancement of demyelinating lesions on MRI.
Exclusion criteria:
No allogenic transplant or gene therapy prior.
Availability of a willing 10/10 HLA-matched sibling donor.
Use of statins or Lorenzo’s oil (must discontinue if accepted in study)
There is a lot more information at the above links.
To talk directly to a the patient coordinator call Tara O’Meara at 617-797-2555 or e-mail