Beyond Batten Disease foundation announces formal sponsorship of Care Beyond Diagnosis in effort to establish Clinical Management Guidelines for CLN3 Disease. 

Austin, TX – Raleigh, NC, September 27, 2021 – Beyond Batten Disease Foundation (BBDF) is pleased to announce a sponsorship agreement with Care Beyond Diagnosis (CBD) to establish internationally validated management guidelines for CLN3 Disease, an ultra-rare, fatal neurodegenerative genetic disorder.

Guidelines which have been methodically and transparently developed can both establish and improve upon a standard of care for patient management. Evidence and consensus-based recommendations for care can improve access to effective management and improve the reimbursement process by recognizing medical necessity.

“Having the support of BBDF is a huge achievement for our team. Obviously, the financial commitment is important for us to keep the program going, but looking ahead towards the publication of the guidelines, BBDF will be instrumental in dissemination of the guidelines into family/caregivers’ hands” says Care Beyond Diagnosis Co-CEO, Jeffrey Donohue. “Engagement from the patient community is a necessity in guideline development.”

President and CEO Mary Beth Kiser says, “BBDF has been keenly interested in guidelines for the CLN3 community for many years.  Thanks to the work of Care Beyond the Diagnosis we are able to meet that need and know this will be a valuable resource for our dedicated community of physicians.”

The development program which began in October 2020 is on track to be completed by year end – 2022 ready for submission to a medical journal. CBD predicts a publication will be available for review by Q2 2023.

The effectiveness of clinical guidelines depends on editorial independence, transparency of process and a validated design. CBD organizes its guideline programs in accordance with the Appraisal of Guidelines for Research and Evaluation’s AGREE II Tool, a 23-point assessment used to assess clinical guidelines.

In the rare disease community, research is by nature, is lacking. A guideline development program identifies evidence gaps in existing literature, but through a Modified Delphi Consensus Program, generates new evidence where those gaps exist.

Guidelines for Rare Disease is currently an unmet need within the community. With over 7,000 rare diseases and a vast majority currently without a disease altering therapy, guidelines can offer caregivers and clinicians a tool to provide patients with the highest QoL possible.

CBD published guidelines for CLN2 Disease in April of 2021. The program was assessed by independent reviewers according to the AGREE II Tool and received a 5.7 out of 7, according to Donohue, was the highest assessment for a rare disease guideline publication.

About Beyond Batten Disease Foundation: Beyond Batten Disease Foundation (BBDF) is the world’s largest non-profit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten disease. Since its inception in 2008, over $35 million has been invested in research by leveraging donations, co-funding and strategic partnerships. BBDF is spearheading a unique, coordinated strategy that incorporates independent scientific resources and collaborates with related organizations to drive research in CLN3 disease. More information can be found at

About Care Beyond Diagnosis: Care Beyond Diagnosis (CBD) is a non-profit organization dedicated towards supporting patients, families and communities following a rare disease diagnosis. CBD has a vision for improved patient-outcomes through both a  small scale, patient specific approach and through large scale, internationally collaborated Rare Disorder guideline development programs.