News

Yeast models of Batten disease and other lysosomal storage disorders could speed up the discovery of treatments by overcoming the restraints of animal models of the disease, a group of researchers argues in the journal Microbial Cell. Their article describes how a research approach that starts in yeast and ends…

The BATCure family survey is now live. The project aims to help develop new therapies for Batten disease by identifying target genes and small-molecule treatments for three types of Batten: juvenile CLN3 (the most common), CLN6 and CLN7. It will also study if existing drugs can be repurposed for Batten.

Early photosensitivity may be a hallmark of ceroid lipofuscinosis type 2 (CLN2) disease, according to the results of a small study. The study “Photosensitivity is an early marker of neuronal ceroid lipofuscinosis type 2 disease” was published in the journal Epilepsia. The neuronal ceroid lipofuscinoses (NCLs), or Batten disease, are a heterogeneous group of lysosomal storage disorders that…

Two 5-year-olds participating in a trial evaluating the first treatment for Batten disease were reunited at Le Bonheur Children’s Hospital in Memphis after Hurricane Harvey almost suspended the treatment for one of them. Elle Gieselmann and Micah McCorkle are among the few Americans receiving the enzyme replacement therapy. The…

An analysis of the protein profile of the three major forms of neuronal ceroid lipofuscinoses (NCLs) has revealed potential biomarkers for the measurement of short-term response to treatment. The study, “Proteomic analysis of brain and cerebrospinal fluid from the three major forms of neuronal ceroid lipofuscinosis reveals potential biomarkers,”…

Abeona Therapeutics will host an inaugural Research and Development (R&D) Day in New York City in October, briefing investors on its progress in developing gene therapies for rare diseases, including two for Batten disease. The R&D Day program and video webcast, set for on Wednesday, Oct. 11, will begin…

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…

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…