Herman van der Putten, our head of research, also attended two other recent meetings:
One on the lysosomal storage diesease Niemann-Pick type C that took place in the Loire Valley in France. For those interested, please consult the website. To select the presentation you may be interested in, please select "scientist's corner". Then select Loire Valley Meeting. Finally select the presentation(s) of interest to you. Especially regarding clinical development of therapeutics, many lessons can be learned from knowledge gained for NPC. We also encourage you to look at the regular NPC Newsletter written by Frank Pfrieger which is available here.
The second meeting entitled "7th Translational Research Conference for the Management of NCLs" took place in Chicago, USA, beginning of November. You will find the agenda of the meeting below. In brief, speakers were grouped by topics and at the end of each session included a moderated panel discussion. The first session gave updates on different translational journeys and clinical trials for a number of NCLs including CLN1, CLN2, CLN3, CLN5, and CLN7, and lessons learned from other rare neurogenetic diseases. The second session was focused on natural history studies, biomarker discovery and other tools to facilitate clinical care and drug development. The third session was on nontraditional clinical and pathological changes in NCL such as sleep and bowel dysfunction. The second day was devoted to therapeutic strategies including small molecules such as miglustat, currently in a clinical trial for CLN3 disease, repurposing of tamoxifen to treat NCLs including CLN3 and CLN7, developing small molecule chemical chaperones (CLN2), progress in how to make enzyme replacement therapies more efficient, viral mediated gene therapy including novel generations of AAV vectors, antisense oligonucleotides for the treatment of CLN3 disease, tackling rare diseases using tRNA-based therapy and genome editing, and unlocking non-viral gene therapy. For more information on each team we suggest you to contact the speaker(s) directly.