News

Treating a juvenile Batten disease mouse model with antisense oligonucleotides to correct its underlying disease-causing mutation led to less waste buildup in the brain, improved motor skills, and longer survival, a study reports.  These results are a key…

Abeona Therapeutics and Taysha Gene Therapies have entered into agreements concerning ABO-202, an investigative gene therapy for infantile Batten disease. The agreements give Taysha worldwide exclusive rights to intellectual property developed by researchers at the University of North Carolina at Chapel Hill (UNC) and Abeona,…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation and rare pediatric disease designation to BBDF-101, an investigational treatment for Batten disease. Theranexus is developing BBDF-101 — which seeks to slow the progression of the inherited neurological condition — pursuant to…

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a gene therapy being developed by Neurogene for the treatment of CLN7 Batten disease. This marks the second Neurogene gene therapy for Batten disease to receive orphan drug designation from the FDA this summer.

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience. Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children…

A case report has described a rare case of a newborn diagnosed with CLN2 disease, or late infantile Batten disease, after a genetic analysis confirmed a disease-causing mutation in the TPP1 gene. The case-study, “A Case with Neonatal-onset Type 2 Neuronal Ceroid Lipofuscinosis: A Novel Mutation,” was published in the…

The University of Rochester Medical Center (URMC) hopes to parlay its new designation as an Intellectual & Developmental Disabilities Research Center (IDDRC) into scientific advances for conditions such as autism and rare disorders such as Batten disease and Rett syndrome. Bestowed by the National Institute…