Ana de Barros, PhD, managing science editor —

The inaugural International Batten Disease Awareness Day was launched on June 9 under the theme “Come Together.” It became a milestone to increase the global reach of social media and unite members of the Batten disease community, while building awareness about the disease and the need for clinical research. The initiative…

A 24-month campaign to raise $6 million toward curing juvenile Batten disease got a $70,000 pop in February, the Beyond Batten Disease Foundation announced in its March newsletter. The Be Project fund-raising effort, which started in 2016, is about $3.7 million, or two-thirds of the way, toward its goal,  according to…

The inaugural issue of the the CRISPR Journal published an article focusing on the potentials of CRISPR gene therapy in Batten disease. In the study “CRISPR-Cas9-Mediated Correction of the 1.02 kb Common Deletion in CLN3 in Induced Pluripotent Stem Cells from Patients with Batten Disease,” researchers from the…

BioMarin recently announced the launch of RARE Scholars, an annual scholarship program for students with certain rare diseases, including Batten disease, who demonstrate strong academic performance, leadership skills, and participation in school and community activities. The program is designed to assist high school seniors, graduates, current postsecondary undergraduates, or…

The New York Stem Cell Foundation and the Beyond Batten Disease Foundation are making juvenile Batten disease stem cells available to researchers. At the moment the collection consists of stem cells from 24 people who have the disease or are carrying the CLN3 gene mutation associated with it. Some of…

Brineura (cerliponase alfa), marketed by BioMarin Pharmaceuticals, was given the WORLDSymposium 2018 New Treatment Award in recognition of its achievements in treating lysosomal diseases, including Batten disease. Brineura slows the decline of children’s ability to crawl or walk, and is the only therapy approved by the U.S. Food and Drug…

A potential gene therapy to treat infantile Batten disease, ABO-202 by Abeona Therapeutics, has been designated an orphan drug by the U.S. Food and Drug Administration (FDA) to help advance its development in clinical testing. Also known as infantile neuronal ceroid lipofuscinosis (INCL), infantile Batten disease is a rare lysosomal storage disease…

The Beyond Batten Disease Foundation has started a Be A Hero campaign to raise $1 million and public awareness about the form of the disease that strikes youngsters. Craig and Charlotte Benson founded the organization in 2008 after doctors diagnosed their then-6-year-old daughter Christiane with juvenile Batten disease. Now 15,…

Brineura (cerliponase alfa), the first therapy approved to treat children with Batten disease, received the 2017 “Best of What’s New” award in the health category by Popular Science, a science and technology magazine. Brineura is BioMarin’s therapeutic product developed to treat the underlying cause of tripeptidyl…

Two parents in Springville, Utah, lost three children in three consecutive days this summer to Batten disease, also known as neuronal ceroid lipofuscinoses. According to The Washington Post, Christopher Chappell, 20, Elizabeth, 19, and James, 15, passed away on a Friday, Saturday, and Sunday, respectively. Their joint obituary…