Cure Mucolipidosis

10/27/2026

🔬 OUR RESEARCH JOURNEY: The long road of peer review

Progress towards future treatments isn’t always visible in a laboratory. Sometimes, it means patiently waiting for the scientific world to catch up with the work already being done.

Our team has written a major review article bringing together what we know about mucolipidosis II/III and what’s needed to move towards future therapies. It’s been under peer review for several months, and we’re waiting for the next step.

Every piece of research matters. Every discovery builds knowledge. And every step brings us closer to our goal. 💙💛

Thank you for standing with Cure ML and helping us keep this important work moving forward.

🌐 curemucolipidosis.org

10/24/2026

🌟 A PROMISING STEP FOR THE RARE DISEASE COMMUNITY! 🌟

For families living with rare diseases, the journey towards treatment can be long, complex and filled with uncertainty. That’s why this announcement from the FDA is so important.

A new Rare Disease Framework Initiative aims to rethink how treatments are developed for rare diseases, recognising that every condition is different and that traditional approaches do not always fit the realities of very small patient populations.

💙 The initiative places greater emphasis on the voices of patients and families, innovative science, and regulatory approaches that reflect the unique challenges of rare diseases — while maintaining rigorous safety standards.

For the Mucolipidosis community, where families are waiting for treatments and researchers are working towards gene therapy, progress like this matters. Developing treatments for ultra-rare conditions requires scientific innovation, collaboration and regulatory pathways that recognise the realities these families face.

We welcome efforts to put rare disease patients and families at the centre of the conversation. We hope this initiative will help create a clearer path forward for promising therapies and bring renewed hope to communities who have waited far too long.

Every step forward matters. Every voice matters. And we will keep pushing for a future where rare diseases are not left behind. 💙

🔗 Read the full announcement:
https://www.hhs.gov/press-room/fda-launches-rare-disease-framework-drug-development.html

The U.S. Food and Drug Administration, HHS, announced the Rare Disease Framework Initiative

10/20/2026

🔬 Research update: Why timing matters

The team’s early findings suggested that treating at around four weeks of age may be too late. So they changed direction and moved treatment much earlier.

That’s research in action — learning from the data, adapting the approach and working towards better outcomes for children with Mucolipidosis. 💛

Help fund the next stage of research: curemucolipidosis.org

10/16/2026

John brings a strong commitment to our ML community and the vision that drives us all: turning research into real hope for families affected by mucolipidosis.

As he steps into this leadership role, John will help guide Cure ML’s mission to advance research, support families and move closer to treatments.

We’re delighted to welcome John as President and look forward to the next chapter of Cure ML under his leadership. 💙

10/13/2026

🧬 What if the secret to a better gene therapy lies in using the gene’s own natural switch?

In our latest research story, we look at how the team is refining the gene therapy tool itself—not just the treatment. By using GNPTAB’s own natural promoter, researchers hope to give cells better control over how the protein is produced.

It’s another important step in the careful process of developing a potential treatment for Mucolipidosis.

💛 Help fund the next round of research: curemucolipidosis.org

09/15/2026

🐭 Want to know what’s really happening behind the scene?

Sometimes, progress looks like… more mouse cages.

And every one matters.

Donate today keep our research moving forward
curemucolipidosis.org

09/08/2026

🧬 **NOW THIS IS PROGRESS!**

**The first results are in — and they’re encouraging. 💛**

✅ The first newborn-treated pups stayed healthy through weaning.
🐭 **24 more newborn pups have now been treated.**
🔬 And the team is watching closely as they grow.

**Small steps. Real research. One pathway forward.**

Help us keep it moving. 💛
**curemucolipidosis.org**

08/25/2026

Our last post shared one of the biggest lessons from the research:

Timing matters. ⏱️

Now comes the next step.

The researchers have taken that lesson into the lab and made a bold decision — they’re treating the mice much earlier, during the newborn period.

Why?

Because if Mucolipidosis α/β begins its earliest damage in the first days and weeks of life, waiting may mean waiting too long.

So the team is moving earlier, aiming to get ahead of the disease before its key features take hold.

It’s a harder approach.
It’s a bolder approach.
And we believe it’s the right direction.

Help us keep moving forward. 💛
www.curemucolipidosis.org

08/18/2026

🧬 OUR PATHWAY TO THERAPY

What does it take to move from an idea in the lab towards a potential therapy?
It happens one discovery at a time.Over the coming weeks, we’ll take you behind the research and share the latest steps in our journey towards gene therapy for Mucolipidosis α/β.

And our first lesson is simple:

Timing matters. ⏱️

The researchers are now treating the mice much earlier — during the newborn period — to see whether getting ahead of the disease can change the outcome.

It’s preclinical research. There are no promises or shortcuts. But every discovery brings us one step closer.

Come with us on the pathway to therapy. 💛

Your support makes this research possible:
www.curemucolipidosis.org

08/04/2026

Every tiny flash of a firefly tells a remarkable story. ✨

What began as one of nature's most beautiful displays is now helping researchers answer one of the biggest questions in gene therapy: Did the gene switch on, and how strongly is it working?

Donate and help us progress this work. www.curemucolipidosis.org