Exciting news! We are thrilled to announce a collaboration with a fellow MCOPS12 patient family based in Australia who have started a non-profit organization, A Cure for Sienna to further support MCOPS12 patients and their families! Together, we are able to co-finance the natural history study for MCOPS12 patients!
Thanks to their incredible fundraising efforts and the support of all of you, we are now able to move forward with this important study!
Why is this Natural History Study so important?
Natural history studies collect health information from patients over time to better understand diseases and evaluate how they progress. By collecting comprehensive medical data from each individual diagnosed with #MCOPS12, we can evaluate the effectiveness of potential therapeutics in clinical trials in the near future.
Once the pre-clinical development of the ASO therapy and the drug repurposing study are finished, thanks to the Natural History Study, we will have tools to measure the effectiveness of those drugs!
Essentially this Natural History Study is a KEY fundamental step for future drug development and treatments for ALL MCOPS12 PATIENTS!
We are beyond grateful to be working with such a dedicated and passionate family and can’t wait to further combine our efforts to support MCOPS12 Patients and their families!
Stay tuned for updates on our progress!