🙌 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 the first step, the design and establishment of a proof of concept for this #ASO therapy to determine if this is a truly viable treatment option for ALL MCOPS12 Patients!
Since we have already signed a contract with Everlum Bio, a rare disease lab who will design and then produce a shortlist of different Antisense Oligonucleotides (ASOs) to determine which ASO is most effective in de-activating the defective gene that causes MCOPS12, we are that much CLOSER to potentially making things like communicating basic needs, sitting, and walking actually POSSIBLE for MCOPS12 Patients!
In the upcoming months we will begin the development of the ASO therapy! We hope to receive the first results within the next 5 months and will keep you all updated with our progress! In order to receive updates on our latest news sign up here: https://rarbmutation.org/blog2/ .
In the next couple of weeks, in parallel to the development of the ASO proof of concept, we plan to turn our efforts towards our second treatment path, creating a Natural History Study! This generally consists of collecting data to describe and understand the disease, which is a prerequisite for testing a new drug in patients.
Please stay tuned to our GFM page, social media, and our website for information on our upcoming campaign for this treatment path!
Please continue to SHARE our Campaign: gofundme.com/f/seeking-a-cure-for-simon
FOLLOW us on social media @curemcops12
TELL your friends and family about our cause!
Thanks again for ALL of your SUPPORT! 💙