There is a bio company, Regenexbio, that has a gene therapy ready to go but the FDA won’t approve the clinical trials. This company has used this type of gene therapy on another neurological disease very similar to Battens disease (called Spinal Muscular Atrophy – or SMA for short) and have had great results with it and have extended the life of children by doubling their life expectancy and still thriving.
The FDA denied the clinical trials for 4 reasons: (1) They want this performed on an adult with CLN2 – which is extremely rare because life expectancy is usually 7-12 years of age with this disease. (2) They want more animal testing. (3) They don’t believe it will be one-shot-and-done. (4) They refuse to look at any of the research on the success of the same therapy on SMA patients. Which is a very similar to CLN2, but worse because the life expectancy of a child born with SMA is 2 yrs of age. They have children who participated in that clinical trial that are now living at 4 and 5 years of age. Proving that it does work.
This therapy isn’t a cure, but it can put our children into a long term remission. We need this so they can stay healthy until they find a cure. That’s the short version.
This petition below was started by a parent with a CLN2 child. We are begging he FDA to start these trials so we can try to save our children before it’s too late. We are in a race against time with this horrific diesase and the longer they prolong it, the more our children degenerate even while on the Brinuera treatment.
