Sponsored Research

Your generosity at work

As a foundation, we are incredibly blessed to have a wonderful community of support embracing our mission and cause. With each and every thoughtful donation, your dollars are put to work funding a variety of important projects. Ranging from therapies for a cure to improving quality of daily life, our goal is to diversify and attack the problem from multiple angles, while being guided by the brightest scientific minds in the rare disease space.

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Below you will find a list of currently funded projects and a brief description of each. Thank you for your support.

Identifying Potential Molecular Therapies for the Treatment of CLN3

Weimer Lab, Sanford Research

 

Antisense Oligonucleotides for the Treatment of CLN3

Dr. Michelle Hastings, Rosalind Franklin University

 

Characterizing the Molecular Function of CLN3 to Identify Novel Therapeutic Targets

Dr. Stephane Lefrancois, Centre Armand-Frappier Sante Biotechnologie

 

Defining Peripheral and Enteric Nervous Disease in Juvenile Batten Disease

Dr. Jonathan Cooper, Washington U. School of Medicine

 

Characterizing Sleep Dysfunction in Individuals with NCL Disorders

Dr. Heather Adams, University of Rochester Medical Center

in Collaboration with the BDSRA

 

Gene Therapy for Children with Pre-Existing AAV Antibodies

Dr. Kathrin Meyer, Nationwide Children’s Hospital