Welcome to the Batten Disease CLN1 Registry
Welcome to the Batten Disease CLN1 Registry from Collaborations Pharmaceuticals, Inc. We are a small pharmaceutical company that has is developing an enzyme replacement therapy for this disease. The objectives of Batten Disease CLN1 registry is to:
- Identify families with children currently living with CLN1.
- Obtain basic contact information on the primary caregiver and disease history.
- Assist in recruitment for future natural history and clinical studies.
- Analysis of the deidentified questions for future publication.
What is a registry?
A rare disease registry provides a means to capture information from patients and their families to help in preparation for a future clinical trial. In general, such registries are used to help connect patient/families with companies and clinicians, enable the understanding of the natural history (how the disease develops), identify outcomes of the disease, while also providing support to help understand the genetic, molecular physiological basis of disease. Such databases provide a starting point to help evaluate potential treatments.
Goals of registry
We know this rare disease is ultra-rare and we need to find as many patients as we can to increase awareness of the disease with regulators and clinicians. We see this as a first small step to help find all the CLN1 patients around the world. We need to find these patients so we can ultimately bring a treatment to them.
How does a registry work?
You can register at the website to provide your basic contact information, provide your informed consent before providing answers to a small number of questions. We collate the deidentified information and learn about the complete patient to assist us with decision making for future clinical trials. Analysis of the basic deidentified data may provide new insights for us that could help with patient care or suggest areas where we need to develop further treatments. We can use the registry to inform you of upcoming webinars to update you on our research and progress.
Registration
If you are the parent / guardian of a child diagnosed with CLN1, and you would like to opt in to register please register here. Your data will not be shared with anyone else without your permission. You can opt out at any time.
Useful links
Collaborations Pharmaceuticals Website: https://www.collaborationspharma.com Our paper on CLN1 ERT: - https://www.jci.org/articles/view/163107