"Your son has Duchenne muscular dystrophy."

300,000 families. One devastating reality:

• Boys lose the ability to walk by age 12
• Most don't see their 30th birthday
• Current gene therapy "treatments" cost $3.2M and barely work

Myosana is the ONLY company delivering the complete, full-size dystrophin protein directly where it's needed. While others fumble with broken approaches, we've engineered the breakthrough the entire industry has been searching for.

IT'S PERSONAL.

Our Board Member's son has DMD. This isn't just business, it's a father's mission to save his child and 300,000 others.

THIS IS REVOLUTIONARY SCIENCE.

Complete protein delivery. Heart muscle treatment. Stem cell repair. Game-changing results.

• Muscles get weaker over time because they're missing a crucial protein called dystrophin
• Boys usually need wheelchairs by age 12 as their leg muscles fail
• Hearts and lungs weaken because they're controlled by muscle too
• Most don't live past their 20s without treatment

Right now, there's only one gene therapy available - it costs $3.2 million per child and barely works. Most families can't afford it, and even those who can see little improvement.

Here's the key insight that everyone else missed:

Think of dystrophin like the frame of a house. Current treatments try to fix the house with a tiny, incomplete frame that's missing most of its parts. That's why they don't work well.

Myosana is the ONLY company that can deliver the complete, full-size frame.

What Makes Us Different

High Costs and viral treatments that deliver only partial protein and have severe side-effects. We deliver the protein safely and repeatedly at accessibly affordable pricing. 

Every muscle cell has special protein "doors" called GLUT4 that only open in muscle cells. We've figured out how to use these doors to deliver our treatment directly where it's needed, like having a key that only opens muscle cell doors.

Why This Matters:

• Gets treatment exactly where it needs to go
• Avoids affecting other parts of the body
• Uses lower doses because we're not wasting medicine
• Much safer approach

This platform is designed to treat Duchenne muscular dystrophy (DMD) by delivering the full version of the dystrophin gene directly into muscles, including the heart, without using viruses. It uses special antibodies to target muscle cells, where it restored dystrophin in DMD mice, reduced muscle damage by 85%, and improved muscle strength. Unlike other approaches, it also works in muscle stem cells, which means it can support long-term muscle repair. The team is now close to selecting the final version for human trials.

Our Laboratory Breakthrough

We're the first and only company to achieve something no one else has:

• Full protein delivery to muscle cells throughout the body
• Stem cell treatment - we can fix the muscle stem cells that repair muscles
• Heart muscle treatment - addresses the heart problems that kill most DMD patients

Why We Believe We Can Win

Dr. Nick Whitehead (CEO and CSO) - 20+ years of muscle research. Discovered how to use GLUT4 as our delivery system.

Dr. Stanley Froehner (Chairman of the Board) - Has studied this disease for over 20 years. Attends every major DMD family conference because he's committed to the community.

Dr. Matt Lumley (Chief Medical Advisor) - Former Moderna and Pfizer executive. His own son has DMD. This isn't just business, it's personal.

Trusted by the Community

The biggest DMD organizations have invested in us:

• Parent Project Muscular Dystrophy (PPMD)
• CureDuchenne
• Muscular Dystrophy Association (MDA)

These organizations evaluate dozens of potential treatments. They chose us.

Size of the Opportunity

• 300,000+ boys worldwide need treatment
• $4+ billion market for effective DMD therapy
• Current treatment costs $3.2 million per patient
• Growing market as awareness increases

Why Now is the Perfect Time

1. Current treatment is failing - families are desperate for something better
2. Gene therapy is accepted - doctors and insurers understand the value
3. We have the breakthrough that others missed
4. Experienced team knows how to get treatments approved

Beyond DMD: Platform for Multiple Diseases

Our technology doesn't just work for DMD. The same approach can treat:

• Limb-Girdle Muscular Dystrophies and X-linked Myotubular Myopathy (other muscle diseases)
• Various heart muscle diseases
• Other genetic muscle conditions

One breakthrough technology = multiple ways to help patients and create value

What We're Building

Myosana’s development plan spans three phases: Phase 1 (next 2 years) will focus on early clinical trials to test safety in boys with DMD and measure protein levels in muscles; Phase 2 (years 3 and 4) will expand to larger studies comparing results with current treatments to show improved strength, mobility, and readiness for approval; and Phase 3 (years 5 and 6) will complete final approval studies and launch the treatment worldwide, bringing hope to thousands of boys and their families.

Why This Is Huge

Gene therapy and rare disease treatments have historically achieved billions in sales, with first-to-market therapies often dominating their field and commanding premium prices.

Our Advantages:

Myosana stands out as the only company offering a complete solution, backed by an experienced team committed to success, driven by personal motivation, and supported by the Duchenne community from day one.

What Success Looks Like

Today: A DMD diagnosis is devastating news. Family life is permanently altered.

Q: How do I know this will work?

A: We have scientific proof that even tiny amounts of the full protein dramatically improve patient outcomes. We're the only company delivering the full protein.

Q: What if other companies develop something similar?

A: We have a 20+ year head start in understanding this disease, plus patent protection on our approach. Other companies are still stuck trying to fix the broken virus approach.

Q: When will patients get treated?

A: Clinical trials are planned to start soon. If successful, treatment could be available in 5 to 6 years - much faster than starting from scratch.

Q: What's my potential return?

A: Gene therapy companies often sell for billions. Rare disease treatments can generate massive returns.

Q: Is this safe?

A: Our approach is potentially much safer than virus-based treatments because we avoid immune reactions and can use lower doses with precise targeting.

TAKE ACTION TODAY

Ready to be part of the solution?

Between a shortened life in a wheelchair and a whole life of possibilities.

Between "there's nothing we can do" and "we have a cure."