A Clinical Research Study for People with Primary Ciliary Dyskinesia (PCD)
The goal of this study by Recode Therapeutics is to find out whether the medicine is safe and may work as a potential new treatment option for those who have PCD due to mutations in the DNAI1 gene. This means it will only work on patients who have an error or "mutation" in the gene called DNAI1 (see our genetics page for more information: Genetics and PCD)
This trial will be a step towards a precision genetic medicine for the treatment of people living with PCD, of which there are currently no approved treatments.
The programme is the result of many years of work and offers the opportunity to alter the course of disease for many people living with rare genetic disorders.
This trial is currently underway at the Royal Brompton Hospital. If you are interested in finding out more, please head to the dedicated Recode Therapeutics PCD page, or chat to your local PCD team about the study: Recode PCD Study Page