The Blog

What is Rare Disease Day? This year, something truly special is happening. Rare Disease Day, a global movement dedicated to shedding light on conditions like #MCOPS12, is just around the corner. Picture this: February 29th, the rarest day of the year, all because of it being a leap year. Since its launch in 2008, Rare Disease Day has become a rallying point for an international community committed to tackling the hurdles faced by individuals living

Happy New Year! 🎉 As we bid farewell to 2023, let’s take a moment to celebrate the incredible milestones we’ve achieved together. From initiating the MCOPS12 Natural History Study to funding the proof of concept for our ongoing ASO therapy development, we’ve been on an amazing journey. This year, we successfully completed our first drug repurposing study and are currently fundraising for a groundbreaking second study testing 5000 drugs. In the spirit of growth and

Here’s to another week of incredible progress and our heartfelt gratitude! We extend our deepest thanks to the extraordinary community that has come together to support the MCOPS12 cause. With 133 donations and a total of $13,919 raised, your generosity is not just a number—it’s a beacon of hope for the future of MCOPS12 kids. Gratitude to Our Champions: A special shoutout to each donor and every individual who has shared our mission far and

We are thrilled to celebrate the incredible generosity of our 97 champions for a cure who, in just a few short days, have brought us just over $11K! Your support is a beacon of hope for MCOPS12 kids, but our journey is far from over. The Progress So Far: In the spirit of gratitude, we want to extend our heartfelt thanks to those who have become true champions for a cure. Your donations have propelled

We’re excited to kick off our end-of-year campaign in collaboration with A Cure for Sophia and Friends on Classy, and we need YOU to be a part of this impactful journey! Today, we’re not just fundraising; we’re rallying for change, aiming to fund a second drug repurposing study addressing the movement disorders faced by #MCOPS12 patients. Our Goal: $100,000 USD – Paving the Way for Life-Changing Treatment! Imagine the power in your hands to directly

As the holiday season fast approaches, it’s easy to get caught up in all the hustle and bustle of holiday shopping and doorbuster deals, taking us away from the spirit of coming together and giving back. That’s exactly why #givingtuesday was created. As a day, to focus back on the spirit of giving and helping others. Here at Cure MCOPS12, Giving Tuesday is more than just a day – it’s a celebration of compassion and

🙌 Exciting News! Partnering with A Cure for Sophia and Friends, we invite you to be part of our end of year fundraising campaign launching on November 28th, on #givingtuesday2023. Together, we aim to fundraise for a pivotal drug repurposing study that holds the potential to transform the lives of #MCOPS12 patients by potentially addressing the movement disorders like hypotonia, spasticity/dystonia experienced by MCOPS12 patients.  The Study: Unlocking the Potential Our goal is to raise

🙌Last week, the Austrian Platform for Personalized Medicine (ÖPPM – Österreichische Plattform für Personalisierte Medizin) held their annual conference, with a focal point of #RareDiseases. Our founder, Reinhard Pell, had the opportunity to present recent progress on our MCOPS12 research, specifically highlighting our ongoing work on a personalized drug development approach using Antisense Oligonucleotides to treat #MCOPS12. Personalized medicine, or precision medicine, harnesses your unique genetic makeup to tailor decisions regarding disease prevention, diagnosis, and

In a world that often celebrates the ordinary, it’s the extraordinary stories that remind us of the incredible strength and resilience of the human spirit. Today, we’d like to share the inspiring journey of Zayd, a member of our MCOPS12 community, whose story showcases determination prevailing over adversity. Zayd’s tale not only demonstrates his courage but also underscores the strength of rare disease communities coming together to provide support. Zayd’s Inspiring Journey Zayd’s story begins

📢Introducing Biswadip Sinha, a dedicated volunteer at Cure MCOPS12 who also works as a Lead of Pharmaceutical Development for Generic Inhalation Products! 🔬 Background and Expertise: Biswadip is a Pharmacist by education and has studied in both India and Germany. He holds a PhD in Pharmaceutical Technology and boasts a wealth of experience in drug product development of various dosage forms, both for new molecules and generic molecules. For the past 13 years, Biswadip has