Gene Therapy Statistics
Both ‘Competing’ Gene Therapy techniques, delivered using an AAV2-hAADC virus vector to either the Putamen or Substantia Nigra & VTA offer improvements to the severe condition Aromatic Amino Acid Decarboxylase deficiency.
Our job at the AADC Trust is to provide support and facts to affected families which are in our possession regarding this invasive but pioneering brain surgery.
With this families are able to make the most informed decision as to whether they take advantage of this treatment which is offering a dramatic improvement to severe AADCd symptoms for many.
Gene Therapy Stats:
More than 20 AADCd children have been treated with Gene Therapy (Putamen) in Taiwan.
More than 6 AADCd children have been treated with Gene Therapy (Putamen) in Japan.
10 more AADCd children in Taiwan are expected to be treated on a Phase I/II trial during the next 18 months.
This trial is ACTIVE but NOT RECRUITING.
8 AADCd children have been treated with Gene Therapy (Substantia Nigra Pars Compacta & Ventral Tegmental Area) in the USA.
15 AADCd children have been treated with Gene Therapy (Substantia Nigra Pars Compacta & Ventral Tegmental Area) in Poland.
Considerably more children in Poland are expected to be treated over the next 18 months on a ‘not-for-profit’ basis.
12 more AADCd children will be treated in the USA over the next 18 months, on the ‘Extended Single-Stage Trial’.
This trial is ACTIVE and RECRUITING.
If you are interested in registering for this trial please click on the link to find out more information. The email contact details are at the bottom of the link page..
A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients (AADC)
National Institutes of Health (NIH)
Information provided by (Responsible Party):
Krystof Bankiewicz, University of California, San Francisco
About The Trust
The AADC Research Trust has advocated on behalf of its sufferers for more than a decade and remains an important 'Worldwide' driving force, inspiring and funding most related research projects, including Gene Therapy.
It remains the only 'dedicated disease support group' available to a global community of sufferers and families. We pride ourselves on providing unbiased information, keeping them up to date and so allowing them to make the most informed decision about their children's futures.
Understanding Gene Replacement Therapy :
Gene therapy is a scientific technique that uses a working gene to treat or prevent diseases.
Gene replacement therapy is a type of gene therapy that uses a “new gene” to replace a faulty or missing gene. This approach attempts to address the root cause of genetic diseases.
Click on the link explore Gene Therapy courtesy of the Columbus Children's Foundation for an explanation of Gene Therapy :