​​ 

 Turning Hope Into Action! 

Our Research Focus:  

​

Thanks to YOU we have raised over $130,000 in the last two years which have allowed us to make substantial progress on our biomarker study in mice models, host our first in person MCOPS12 symposium and get to the final stages of our second drug repurposing study.

 

We now need your help to raise an additional $200K US by the end of our fiscal year in June 2026 to proceed promising candidates from our drug repurposing studies into a clinical trial. To achieve this goal, our steps include two new biomarker studies, a new patient cell model, continuing the MCOPS12 Natural History Study at CHU Sainte-Justine, and co-financing a PhD student for translational sciences.

 

Biomarkers:

In addition to the ongoing mouse-model biomarker study, we will investigate proteins and metabolites in MCOPS12 patients’ blood samples (along with control samples) which are distinctive for disease progression and/or amelioration after a medical treatment. As we shared last year, biomarkers are strongly recommended by health authorities as an additional/secondary endpoint to assess a drug´s efficacy in a clinical trial.  In addition, the study will help us to better understand the pathology of MCOPS12. 

 

New patient cell model and translational science:

Both studies aim for obtaining a holistic picture on the pathophysiology of MCOPS12. We will generate a second patient-derived cell model containing a different type of neurons compared to the initial cell model. Biomolecular studies will identify dysfunctions compared to healthy cells. Both cell models and their readouts will serve as the ideal platform to test established drugs to repair the dysfunction (i.e. perform a targeted drug repurposing).

 

Natural History Study:

We are happy to announce that our natural history study researchers working at CHU Sainte-Justine have agreed to work in conjunction with the company processing our biomarkers so we can keep all our data in one place to further expand on results from both studies.   CHU Sainte-Justine has generously covered the salary for the coordinator of our Natural History study for the past two years along with a large portion of our costs for our first in person conference and will need assistance to continue the study past this year.

 

Your gift will give us the chance to make improved motor and cognitive function a reality for our families around the world!

​

Merci, Grazie, Cheers, Arigato, Tack, Kiitos, Go raibh mile maith agat,

Danke, Thank you

​​​

How You Can Be a Champion:

​​