Gene therapies for children with Batten disease are being evaluated in two clinical trials — one in New York and the other in Ohio. The trials are testing ways to treat two types Batten disease by correcting the genetic defect that causes it. One study is a Phase 1/2 clinical trial…
News
The U.S. Food and Drug Administration (FDA) granted orphan drug status to Polaryx Therapeutics’ PLX-200 in response to a lack of treatments available for late infantile neuronal ceroid lipofuscinosis (LINCL), a type of Batten disease. PLX-200 is a repurposed drug (one that was developed for one indication…
The Charlotte and Gwenyth Gray Foundation to Cure Batten Disease was launched in 2015 by Hollywood producer Gordon Gray and his wife Kristen to advocate and raise funds for research to find a cure for Batten disease. The foundation also supports patient therapies and the purchase of equipment crucial…
Even by the standards of rare illnesses, Batten disease is extremely uncommon, affecting only two to four of every 100,000 births in the United States. That translates into 20 or so American babies born each year with the hereditary illness. Yet no disease is too insignificant for the…
Current therapeutics, including gene therapy, that are based on delivery of TTP1 protein directly into the central nervous system of patients with Batten disease may not be enough to prevent all the symptoms associated with this condition. Using a canine model for the disease, researchers showed that this approach did…
Researchers studying the loss of the CLN5 protein, which causes Batten disease, found that it leads to the reduced production of new fully functional neurons. It also increased neuronal inflammation in mouse models of the neurodegenerative disorder. The study, “Loss of CLN5 causes altered neurogenesis in a childhood neurodegenerative…
A small North Carolina nonprofit organization, Indo Jax Surf Charities, has recently gained attention for its set of free programs to help children with Batten disease and other special needs build self-esteem. Since launching these programs, Indo Jax has been featured in several media platforms, including American Way…
New research shows that patients with infantile Batten disease exhibit significant early disease in the spinal cord and that combined treatment of the spinal cord and the brain has significant therapeutic effects. The study titled, “Synergistic effects of treating the spinal cord and brain in CLN1 disease,” was published…
Gov. Tom Wolf Pennsylvania Gov. Tom Wolf recently signed House Bill 239, establishing the Rare Disease Advisory Council. The bill, was introduced May 24 by state Rep. Marcy Toepel, R-Montgomery County, who said the bill is an outgrowth of contact with people in her district who…
European and U.S. regulators have released a white paper on ways to develop innovative therapies for Gaucher disease that could also apply to Batten disease and other rare children’s disorders. The goal of the European Medicines Agency and U.S. Food and Drug Administration paper is to spur novel approaches to creating treatments.
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