While targeted towards those 17 and under, these trial/studies are promising for the entire community. I have become friends with a lot of parents who have children with the childhood gene who are always bouncing questions off of me and I'm always hopeful these new procedures will be able to help them.
"Long-Term Follow-up Data Show That the 88% of Patients Treated in the Starbeam Study (ALD-102) Were Free of Major Functional Disabilities (MFDs) at Two Years, and Continued to Remain MFD-Free at up to Five Years of Follow-up"
I would like to know whether abcd1 gene can be normally expressed after gene therapy, which is helpful for our patients with amn. My doctor is currently applying for an ethical review and, if approved, will also conduct a clinical trial of gene therapy in ald. I am considering whether to participate.
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AMN and me
Life expectancy.
