Researchers described the natural history of retinal degeneration in a mouse model of CLN7, a form of late-infantile Batten…
Articles by Steve Bryson, PhD
Although electroencephalogram (EEG) and MRI findings may detect early signs of ceroid lipofuscinosis type 2 disease (CLN2), they cannot…
Two young sisters who carry a unique combination of mutations in their CLN8 genes developed a mild, but atypical…
The CLN7 protein that is faulty in a form of late-infantile Batten disease is a chloride channel that regulates…
The benefits of using telehealth services for people with rare conditions, such as Batten disease, include convenience, cost savings,…
Gene therapy safely and effectively eased the signs and symptoms of CLN7 disease, a form of late-infantile Batten disease,…
Taysha Gene Therapies has secured an exclusive option from the University of Texas Southwestern (UTSW) to license worldwide rights…
The U.S. Food and Drug Administration (FDA) has cleared Neurogene’s request to conduct a Phase 1/2 clinical trial to…
Animal models predicted dosing of the enzyme replacement therapy Brineura (cerliponase alfa) for patients with late infantile Batten…
An international team of experts has provided the latest evidence-based recommendations for diagnosis, assessment, management, and treatment…