The Blog

🙌 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

We’re happy to announce that our GoFundMe Campaign is officially LIVE! 🥳 Please find it here: https://gofund.me/a0081c7c ! It has been a long journey to get here, but we’re excited to start fundraising for one of the two treatment paths we’ve identified for MCOPS12 Patients. The first path is ongoing and focuses on testing drugs in disease models to improve the two major symptoms of MCOPS12 patients – their severe and progressive movement disorders and

We’re excited to announce we will be launching a GoFundMe Campaign on the 29 November 2022! Our goal is to ultimately fund the creation of a therapy which uses Antisense Oligonucleotides (ASOs) – which are designed to target the “bad RNA” found in MCOPS12 patients. This therapy would enable us to directly combat the disease at the genetic level, which is critical in halting further progression and improving symptoms caused by this disease. This therapy

Drug repurposing (also called drug repositioning) is the process of investigating new therapeutic purposes for already existing drugs. The drugs of interest may either be commercially available or still in clinical trials – what they have in common is that their mechanism of action is understood, and their efficacy and (potential) side effects in humans are already known. Thus, compared to “conventional” drug development programs using novel drugs, drug repurposing is much faster and cheaper