A Mother’s Mission: Raising Mucolipidosis Awareness
- Jase
- 19 minutes ago
- 3 min read
A Mother’s Mission: Raising Mucolipidosis Awareness
When Rare Means Invisible
Most people have never heard of Mucolipidosis type III (ML3). That’s because only six people in Australia—and about 125 worldwide—are known to live with it. When numbers are that small, conditions like ML3 slip under the radar. They don’t make headlines. They don’t attract major research funding. And sadly, they often don’t get the recognition they deserve.
But for families like Juanita’s, ML3 is a daily reality. Her sons, Damian (19) and Jesse (17), are both living with the painful, life-limiting condition.
What Life with ML3 Really Means
On paper, ML3 is described as causing progressive joint stiffness, chronic pain, and repeated surgeries. But those words don’t fully capture the reality.
Everyday basics become battles. Getting dressed can take an hour because shoulders, elbows, and fingers won’t cooperate. Simple things like tying shoelaces or fastening a button often need help.
Walking is painful. Crossing a room can feel like running a marathon. Hips and knees ache constantly, and climbing stairs isn’t an option—ramps and lifts aren’t conveniences, they’re lifelines.
Independence is stolen piece by piece. Sports, music, carrying groceries—things most young people enjoy—become impossible or out of reach.
The surgeries add up. For many with ML3, it’s not one or two major operations in a lifetime. It can be dozens of surgeries—hip replacements, spinal corrections, procedures to ease pain—each one followed by long recoveries, scars, and new limitations. Broken bones are common too, because fragile joints and weak connective tissue give way under normal strain.
And the hardest part? It doesn’t stay the same. What Damian and Jesse can do today may be gone tomorrow. That slow, steady loss of independence is just as heavy as the physical pain.
Why Rare Diseases Struggle for Attention
Big pharmaceutical companies typically invest in treatments that affect large populations—it’s where the profit is. With ML3 affecting so few, commercial interest is virtually non-existent. That leaves families carrying the burden of driving awareness and funding.
This is part of a wider issue: rare diseases as a group aren’t rare at all. Collectively, over 300 million people worldwide live with one. Yet because each individual condition is so small in numbers, research often lags decades behind more common illnesses.
A Global Effort for a Genetic Cure
Juanita and her family have joined forces with others worldwide to raise $1 million AUD for gene therapy research. The science is already in place, and clinical trials could begin as soon as 2028. If successful, it could become the first-ever genetic cure for Mucolipidosis.
This campaign isn’t just about Damian and Jesse—it’s about proving that even ultra-rare conditions are worthy of hope, attention, and investment.
Why Awareness Matters
Awareness is more than sharing a hashtag. It’s giving families visibility. It’s recognising the courage of young people like Damian and Jesse. And it’s creating momentum for funding, clinical trials, and—ultimately—a cure.
Without awareness, rare diseases remain invisible. With it, they stand a fighting chance.
How You Can Help
Donate: 100% of donations go directly to research, and all contributions are tax-deductible.
Share: The more people know about ML3, the faster the path to treatment.
Follow: Stay updated via Team Damian Jesse Rose on Facebook.
🔗 Donate here: cure-ml-australia.raiselysite.com
Together, we can give Damian, Jesse, and others like them the future they deserve.
The Ripple Effect of Rare Disease Research
There’s another powerful reason to support research into ML3: science never stops at one discovery. Raising Mucolipidosis awareness doesn’t just shine a light on one ultra-rare disease—it helps push science forward in ways that ripple out to many others.
When researchers unlock the mechanics of a rare condition like ML3, that knowledge can spill over into other areas. Advances in gene therapy, for example, don’t just benefit one disease. The same techniques may be adapted to help people with other genetic disorders, degenerative conditions, or even common illnesses that affect millions.
So every dollar raised isn’t just pushing towards a cure for Damian and Jesse—it’s helping to build scientific tools and knowledge that could improve lives far beyond the ML3 community.
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