Taysha Gene Therapies has recently released the attached letter on their CLN1 Gene Therapy program to the Batten community. You can read the letter here!
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 [...]
Model systems are an important part of disease research. These model systems can range from simple single cellular systems to larger animal models. The BDSRA has helped to fund many of these animal models to [...]
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 [...]
Abeona is eager to continue its commitment to the Batten community as they focus on bringing the potential CLN3 gene therapy, developed by Tammy Kielian, Ph.D. and her team at the University of Nebraska Medical [...]
Tammy Kielian, Ph.D., professor in the UNMC Department of Pathology and Microbiology, has been named the 11th UNMC Scientist Laureate.
The award is the highest honor UNMC bestows to researchers. Dr. Kielian was honored along with 21 other researchers recently named UNMC Distinguished Scientist, New Investigator and Research Leadership award winners for 2016. “Tammy Kielian [...]
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 [...]
Ryan D. Geraets, Seung yon Koh, Michelle L. Hastings, Tammy Kielian, David A. Pearce and Jill M. Weimer Abstract The Neuronal Ceroid Lipofuscinoses (NCLs) are a family of autosomal recessive neurodegenerative disorders that annually affect [...]
Rutgers Scientists Develop Therapy for Fatal Childhood Disease The R&D Council of New Jersey has honored two Rutgers University professors with its Edison Patent Award for discovering the cause of a rare but devastating and [...]
One of the most important steps toward finding effective medical treatments for Batten disease and advancing clinical research is to map out the ways in which the forms of the disease affect patients: the symptoms [...]
Bridget Kennicott was born on November 11, 2004, to proud parents Sara and Dave and big brother Harrison. The Carpentersville, Illinois family felt complete, with their energetic little daughter soon known as “Miss Busy B.” [...]