Author Archives: Marisa Wexler MS

Seizures Were Only Symptom of Juvenile Batten in Girl, 13: Report

A 13-year-old girl experiencing unexplained seizures and no other apparent symptoms was ultimately diagnosed with juvenile Batten disease, according to a recent report. This “is probably the first case report of [juvenile Batten disease] with only seizures as presentation,” the researchers wrote. Titled “Batten’s Disease: A…

Europe Awaits Proposed New Framework for Sharing Health Data

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

CLN5 Gene Therapy Trial Now Enrolling Children at New York Site

Children with CLN5, a type of late infantile Batten disease, are now being recruited for a first-in-human clinical trial testing NGN-101, Neurogene‘s experimental gene therapy for the rare neurological condition. The trial will be conducted at the University of Rochester Medical Center (URMC), in upstate New York, the company …

CLN5 Gene Therapy Trial to Start Early Next Year in Rochester

Enrollment in a clinical trial testing NGN-101 — an experimental gene therapy for CLN5, a type of late-infantile Batten disease — is expected to start early next year at the University of Rochester Medical Center (URMC). “This trial will move research forward in developing a potentially disease-modifying treatment for…

Actelion to Help Develop BBDF-101 for Juvenile Batten Disease

Beyond Batten Disease Foundation (BBDF) has struck a deal with Actelion Pharmaceuticals to help further the development of BBDF-101, an investigational treatment for juvenile Batten disease. Under the terms of the new agreement, Actelion (a Janssen Pharmaceutical Company of Johnson & Johnson) will provide a component of…