Research and surveys
UK sites looking to recruit individuals with Gorlin syndrome for Patidegib Phase III Trial
Are you interested in participating in this Phase III trial? This is a global study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with Gorlin syndrome. For more information about...
Do you want to take part in shaping the future?
Courtesy of Camilla Krogh Lauritzen, Chief Patient Officer, Rare Diseases, LEO Pharma At LEO Pharma, we know that people living with rare diseases are experts in their own right: They are indeed disease experience experts. We also appreciate the relevance for us to...
Patidegib Phase III Trial – EU Website is Live
The EU website for the Pelle-926-301 study is now live. You can see it at www.global.gorlinstudy.com Study sites in France, Spain and Germany are now active. Clicking on the respective flag will launch the content in that language. It is anticipated that other sites...
GORLIN UNCOVERED – Summary of survey results
An on-line survey ran from 29/7/18 to 4/7/18. Participants could volunteer if they were 18 or over and had Gorlin syndrome. We asked participants to respond for themselves. There were 39 responses, including one form an individual who said she was not diagnosed with...
Germline Genome Editing Research
Germline Genome Editing (Genetic Editing) is a technique through which parts of DNA in an egg, sperm or embryo can be added, removed or replaced. It can be used to prevent hereditary diseases by ensuring DNA sequences known to cause specific conditions are not passed...
Screening for the patidegib phase III trial
What is screening? Screening in the process used to see who is suitable to participate in a clinical trial. Screening uses a checklist of inclusion and exclusion criteria. You can read more about these here. The criteria aim to do several things If you go for...
Understanding Clinical Trials
Why do clinical trials? Over the past couple of years, clinical trials are coming on line for people with Gorlin Syndrome. Clinical trials are usually used to test new treatments (for example, patidegib) or older treatments that are being tried for the first time in...
Gorlin Syndrome Survey – Potential New Medication
In recent weeks we have been discussion with LEO Pharma regarding the development of a new medication. The purpose of the survey is to find out how individuals would feel about a medication which could prevent basal cell cancers. The proposed medication is in the...
Oxford University Research – Rare Genetic Syndromes
At Oxford University, Dr. Chris Nellaker is working on teaching computers to recognize rare genetic syndromes through photos of patients. He is passionate about making it easier for doctors to help families to identify these syndromes. Dr. Nellaker is using machine...
Research Projects
The Gorlin Syndrome Group is committed to the promotion of research into the causes, effects, treatment and management of Gorlin Syndrome and the dissemination of the useful results. On occasions we are contacted by organisations and medical practitioners wishing...