Clinical News

WORLDSymposium 2021

WORLDSymposium 2021 February 8-12 basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of [...]

Batten Research Learning Lab: Therapeutic Strategies

Below are the most common therapeutic strategies in use for Batten disease What is Enzyme Replacement Therapy? (From Guide to Symptomatic Treatment of Neuronal Ceroid Lipofuscinosis) Enzyme infusion has been proven effective for other lysosomal storage [...]

Meet Our Researchers: Sue Cotman, PhD and Cotman Lab

This month we are excited to highlight the work being done by Sue Cotman Ph.D. and Cotman Lab! Dr. Cotman, what is your current position? Assistant Professor of Neurology, Massachusetts General Hospital, Harvard Medical School [...]


Presentations and posters shared by Batten researchers included: Presentations Alejandra J. Rozenberg: University of North Carolina at Chapel Hill, North Carolina, U.S.A. Early intrathecal gene therapy extends lifespan and improves quality of life in a [...]

Meet Dr. Jon Cooper

This summer Jon Cooper and his family moved from England to Southern California, and we asked him some questions for our Researcher Q & A How long have you been studying Batten disease? I’ve been [...]

Dr. Marco Sardiello

Dr. Marco Sardiello is an Assistant Professor at the Baylor College of Medicine in Houston, Texas.  In its latest funding round, BDSRA’s board approved his proposal:  mTORIndependent Lysosomal Enhancement for the Treatment of Neuronal Ceroid [...]

New CNS President

Dr. Jonathan Mink was recently elected as the President-Elect of the Child Neurology Society.  Dr. Mink is a 2016 BDSRA research awardee and the director of the BDSRA Center of Excellence at the University of [...]

Program to Develop New Therapies for Batten disease

Sparks Children's Medical Research Dr Sara Mole, who has a Sparks-funded project, has recently begun a large programme of work aimed at developing new therapies for Batten disease.  Dr Mole led a consortium of researchers [...]

BioMarin CLN2 Results

“Intracerebroventricular cerliponase alpha (BMN 190) in children with CLN2 disease: Results from a Phase 1/2, open-label, dose-escalation study (A. Schulz et al) The following is reported with our thanks to the BDFA: 87% of children [...]

January 2016 Research News

2016 is going to be a big year for translational research-from bench to bedside, thanks to researchers who have benefited from BDSRA families’ funding efforts.   Additionally, great strides are being made in the gene [...]