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Gene Therapy and Misperceptions about CdLS

We are aware that there have been some independent fundraising campaigns set up to research gene therapy to improve aspects of CdLS. We will always support research that aims to benefit the CdLS community. We also have our own fundraising needs and at this time, our work to support families affected by CdLS, is still very much needed. We are cautiously optimistic that we may see viable therapies, whether involving genes, medications or other interventions as a result of any research efforts at some point in the future. We very much hope that any such research is successful.

We are also aware that some inaccurate information has been circulated about CdLS. Some has been addressed directly but we want to reassure any family affected by CdLS that correct and up to date information about CdLS is to be found on the pages of this website, that of the CdLS Foundation USA and any of the national support groups’ sites, FIND or the World Federation website. We know that some families have circulated behavioural research papers. We have a statement that addresses the correct meaning of these research papers, which can be downloaded here. When asked if CdLS is degenerative, one of our longest serving clinicians answered, "Definitely not! In fact I have observed that individuals with CdLS continue to learn no matter how old they are. No loss of neurologic function or ability to do tasks. At times however there could be depression or anxiety or something that makes the individual prefer to stay at home doing minimal social activities etc." A paper about Executive Function changes has been quoted out of context. A summary of actual findings can be found on this link. There is an updated supplement to Reaching Out on Adolescence and Adulthood, which may be of help on this link.

Several of our professionals have advised on the current state of knowledge regarding CdLS and the possibilities of gene therapy. Our own UK & Ireland Scientific & Clinical Advisory Team (SCAT) released this statement. We also have a joint consensus letter from the USA Foundation and International Scientific Advisory Council (SAC) to families on this link. There is a fascinating piece from Professor Raoul Hennekam from the Netherlands CdLS group on this link. All can be downloaded by clicking the relevant links.

If you have any questions or concerns, please contact our office. info@cdls.org.uk