In expectations of preparing this petition. I would like to express our concerns and be the voice of our children and all CLN2 parents. The FDA has denied access to clinical trials for gene therapy for our CLN2 children. Gene therapy has helped other rare disease patients.

 

Here is A brief explanation of CLN2 Battens Disease:

 

CLN2 disease (Neuronal Ceroid Lipofuscinosis Type 2) is an ultra-rare, neurodegenerative lysosomal storage disease, caused by an enzyme deficiency of tripeptidyl peptidase 1 (TPP1).

 

CLN2 disease affects motor skills, such as sitting and walking, and speech development. This condition also causes the loss of previously acquired skills (developmental regression), intellectual disability that gradually gets worse, and behavioral problems. Individuals with this condition often require the use of a wheelchair by late childhood and typically do not survive past their teens.

 

      This is not the life that our children deserve to live. There’s better treatment for our children, and that’s gene therapy. We have rights as parents and our voices need to be heard. Our children receive enzyme replacement therapy. This produces our children with synthetic enzymes. This is a bi-weekly treatment, some kids have been receiving this treatment for over five years. To be able to have gene therapy offered to our children, would allow many children to live a normal life.

 

     If the FDA can please take some sympathy to listen to the concerned voices of all CLN2 parents, we can make a difference. Life has better options, and gene therapy is the number one step towards it for our children. This is not how any of us predicted for our life to be, and science has proven that it can make miraculous changes. Allowing clinical trials to take place, would be the most ethically responsible and humane thing to do.

 

     Science studies has also shown allowing CLN2 patients to produce natural enzymes, will improve all motor skills and mobility and speech. It is ethically right to allow scientist, to move forward with the clinical trials.

 

      It is also ethically right to want to help others, and not frown upon wanting to provide a treatment that can be extremely beneficial for our children. Scientist have already begun to work on the treatment, and it would be highly appreciated if clinical trial for gene therapy for CLN2 would be provided to our children.

 

    My question in theory is how come the FDA is giving us CLN2 parents a hard time to grant clinical trials for gene therapy? This is life threatening to our children! Time is of the essence, and with such a harmful rare genetic disease, every moment counts.

 

Thank you for taking the time to review this petition.