BALTIMORE, November 18, 2025 —Kennedy Krieger Institute has been awarded an $8.17 million grant from the National Institutes of Health to establish and lead a first-of-its-kind network in the U.S. dedicated to advancing treatments for Batten diseases, a group of rare pediatric neurodegenerative disorders.
The grant positions Kennedy Krieger as the coordinating center for the 14-site Batten Disease Clinical Research Consortium (BDCRC). Under the direction of Dr. Erika Augustine, Director of Kennedy Krieger’s Batten Disease Center of Excellence, the BDCRC will be headquartered at Kennedy Krieger which and the will serve as the scientific and administrative hub for the network.
"The BDCRC represents a major opportunity to close critical gaps in knowledge about Batten diseases and readiness to design and launch clinical trials," Dr. Augustine said. "By uniting leading academic centers and advocacy partners, we are laying the groundwork for meaningful advances."
Batten diseases, also called neuronal ceroid lipofuscinoses, are inherited neurogenetic disorders that often cause vision loss, motor and cognitive decline, seizures, and even premature death. More than a dozen subtypes of Batten disease exist, and most do not have effective treatments.
Kennedy Krieger will become part of the Rare Diseases Clinical Research Network (RDCRN), and work closely with Our Promise to Nicholas Foundation, and Batten Disease Support, Research, and Advocacy Foundation (BDSRA), ensuring patient perspectives are emphasized. Amy Fenton Parker, the President and CEO of the BDSRA Foundation, says this will help guide research priorities.
"This grant is extraordinary news for the Batten community," said Parker. "The BDSRA Foundation is thrilled to collaborate with Kennedy Krieger and the BDCRC to help keep the Batten community's research priorities front and center, while new ideas are pursued toward unmet needs for clinically relevant and effective treatments."
The project is funded by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Institute of Neurological Disorders and Stroke (NINDS), and the National Center for Advancing Translational Sciences (NCATS).
BDCRC sites include Boston Children’s Hospital, Children’s Hospital Colorado, Children’s Hospital of Orange County, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Nationwide Children’s Hospital, NYU Langone Health, Rush University, Sanford Research, University of Michigan, University of North Carolina at Chapel Hill, University of Rochester, and Washington University in St. Louis. A national Batten disease biorepository will also be launched to provide a foundation for future discoveries.
RCDRN will post updates on the project with work beginning soon. Visit our website to learn more about additional Batten disease research underway at Kennedy Krieger Institute.
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نبذة عن معهد Kennedy Krieger
يُعد معهد Kennedy Krieger منظمة غير ربحية معروفة عالميًا، يقع في منطقة بالتيمور/واشنطن العاصمة الكبرى، ويُحدث تأثيرًا في حياة ما يقرب من 30,000 فرد سنويًا من خلال تقديم مجموعة واسعة من الخدمات، تشمل: العلاج الطبي الداخلي والخارجي، وعلاجات الصحة السلوكية والعافية، وخدمات الرعاية المنزلية والمجتمعية، والبرامج المدرسية، إلى جانب التدريب والتثقيف المهني والدفاع عن القضايا المتعلقة بالصحة العصبية. ويقدم معهد Kennedy Krieger مجموعة واسعة من الخدمات للأطفال والمراهقين والبالغين الذين يعانون من أمراض أو اضطرابات أو إصابات تؤثر على النظام العصبي وتتنوع شدتها من المتوسط إلى الحاد. ويترأس المعهد فريقًا من الباحثين الذين يساهمون في فهم نشوء الاضطرابات مع استحداث أساليب تدخل جديدة وطرق تشخيص ووقاية وعلاج مبكرة. يرجى زيارة www.KennedyKrieger.org/ لمزيد من المعلومات حول معهد Kennedy Krieger.
