Author Archives: Steve Bryson PhD

FDA OKs Trial of NGN-101 Gene Therapy in CLN5 Patients

The U.S. Food and Drug Administration (FDA) has cleared Neurogene’s request to conduct a Phase 1/2 clinical trial to evaluate the safety, tolerability, and efficacy of NGN-101, an investigational gene therapy to treat CLN5, a form of late-infantile Batten disease. The therapy uses a harmless adeno-associated virus (AAV)…

Animal Models Can Predict Brineura Doses for CLN2 Patients

Animal models predicted dosing of the enzyme replacement therapy Brineura (cerliponase alfa) for patients with late infantile Batten disease, also called CLN2 disease, a study showed. Scientists said this approach also may be applied to predict cross-species dosing for other similar medicines. “Overall, the approach described here provides…

New Guidelines Issued for CLN2 Diagnosis, Management

An international team of experts has provided the latest evidence-based recommendations for diagnosis, assessment, management, and treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), a study reported.  The study, “Guidelines on the diagnosis, clinical assessments, treatment and management for…