The Blog

🎉Happy #RareDiseaseDay everyone! Today we recognize the millions of people living with #rarediseases and their families, who face unique challenges every day. Rare diseases affect more than 300 million people globally, yet they often go undiagnosed and untreated due to a lack of awareness, research, and funding… Let’s work together so that this isn’t the future for patients with #MCOPS12 . MCOPS12 is an ultra-rare neurological disease that causes severe and progressive movement disorders, developmental

🙌We are happy to announce we are launching our next campaign! We are now able to take the next critical step in finding a therapy for #MCOPS12 patients! In collaboration with the CHU Sainte-Justine Research Centre in Montréal, Cure MCOPS12 will establish and co-finance a Natural History Study for MCOPS12 patients.  Natural history studies collect health information from patients over time to better understand diseases and evaluate how they progress. By collecting comprehensive medical data

Many #raredisease patients and their families face a multitude of obstacles due to various #healthinequities – from challenges accessing and acquiring necessary social and health services to receiving medicines and treatments.  In honor of #rarediseaseday we are highlighting some of the various health inequities that #MCOPS12 patients and their families have faced.  The Pell Family, who live in Austria with their son Simon, have faced many obstacles. One of the biggest issues has been all

🙌 We have an AMAZING announcement to make – we have officially REACHED our goal of 75K!! Words cannot express how THANKFUL we are for all of your support – from helping us spread awareness to making donations – THANK YOU! Each and Every One of You have played a part in making a difference in the lives of #MCOPS12 Patients worldwide! Now that we’ve reached 75K we can get the ball rolling and begin

🎶 On Our 12 Days of Christmas we have TWELVE wishes specific to #MCOPS12 Kids, Their Families, and Cure MCOPS12. We have kids that would LOVE to one day be able to do things that other kids can do… To SIT unassisted, to WALK, to have MORE Independence, to be able to make their own MARK on this world. We have families that want to be able to share their passions and hobbies with their

In just a little over a month we’ve managed to raise almost 65K!! 🙌 We are so VERY grateful to all of you who have supported us so far – Thank YOU! 💙 Our campaign for this proof of concept for the #ASO therapy will go until the end of January and we STILL NEED about 10K… Patients with MCOPS12 and their families face various challenges and difficulties EVERY SINGLE DAY due to the symptoms

Hey Everyone! We have some EXCITING news we’d like to share with you all! We have signed a contract with Everlum Bio, a Rare Disease Lab, that will design and then produce a shortlist of different Antisense Oligonucleotides (ASOs) that will initially be tested in a lab model consisting of Simon’s neurons! During this testing phase, they will determine which ASO is most effective in deactivating the defective gene that causes MCOPS12! BUT in order

🎊We’ve reached over 50K!! Thanks to all of your continued support! 💙 However, we STILL NEED to raise 25K – without the total 75K we won’t be able to fund this potentially LIFE CHANGING proof of concept… We have less than a month to go, Let’s make the impossible, POSSIBLE!   ✨ We are kicking off the holiday season with a #12daysofchristmas campaign! Every day starting on the 25thof December until the 6th of January we will

We can’t believe it – We’re OVER HALFWAY there!! After only two weeks we’ve made such great progress, giving us all so much HOPE that we will be able to develop this proof of concept for the ASO Therapy!  So far, we’ve raised €44,108 – bringing us that much closer to reaching our €75,000 goal – Thanks to ALL OF YOU!  We have about A MONTH LEFT to raise a little more than €30,000  – we believe that we can do

It’s already been a week since the launch of our Campaign, and we can’t believe the amazing start we’ve had! 🎉Thank you to all of you who have donated, shared, and been involved in the campaign thus far – we are beyond grateful for you and what you’ve helped us to accomplish! We have successfully received €34,438 and truly this wouldn’t have been possible without all of you! 💙What does this mean?Well, this amount allows