Ó³»­´«Ã½

Answers to some of the most common questions we receive are listed below as a resource for researchers, physicians, patients, caregivers, and patient advocacy groups. Please feel free to contact us with additional questions. As a note, we cannot offer medical advice or interpret test results.

How can I stay in touch with Ladders to Cures?

There are several ways to stay connected with the Ladders to Cures Accelerator. Please click here to join our L2C Accelerator network. We will keep you in the loop on upcoming events and research highlights.

I’m a rare disease researcher. Where can I find additional resources?

Does the Ó³»­´«Ã½ provide research-based sequencing for patients and families with undiagnosed, suspected genetic diseases?

Please visit the to learn more about patient-driven research studies aimed at discovering the genes underlying rare diseases.

Where can I learn more about rare diseases and connect with other patients and patient advocates?

How can I sign up myself/my loved one to participate in clinical trials?

While our work is intensely focused on understanding the rare genetic diseases that could benefit from our discoveries, we do not run any human clinical trials. As a research group, we work to identify the most promising, pre-clinical projects addressing nodal biology. We predominantly work with cell lines and animal models of human genetic diseases. Our vision is that the resulting research will enable scientists in industry or other clinical organizations to develop safe and effective therapeutics.

You can learn more about how clinical trials work . To search for clinical trials that are relevant to you or your loved one, please visit: . To access a curated list of gene and cell therapy trials, please visit the . Please speak with your healthcare provider before enrolling in a clinical trial.