Do you believe miracles are impossible and out of reach?

Together, we can make one happen for Anwar. ❤️

A life-changing gene therapy could give Anwar a chance to fight muscular dystrophy and change his lifeforever

the Enemy: A Journey with LGMD2D

Imagine waking up one day to realize that time has become your greatest enemy—that you are racing against it while fighting a rare, progressive disease that stole your childhood and now threatens to steal your future.

My life changed forever in June 2021, when I was diagnosed with Limb-Girdle Muscular Dystrophy type 2D (LGMD2D). Since that day, time has no longer felt like something that simply passes. It has become something I constantly race against.

LGMD2D is a rare genetic muscle disease that progressively weakens the muscles responsible for some of the most basic movements of daily life. Things that most people do without thinking—standing up, sitting down, climbing stairs, lifting an arm, walking across a room can become exhausting battles.

When the Simplest Movements Become a Battle

There is a strange kind of grief in having to think about movements that everyone else performs automatically.

I have forgotten what it feels like to walk without fear.

I have forgotten how people sit down without pain, without calculating the movement, without wondering whether their muscles will cooperate.

I watch people walk, rise from chairs, climb stairs, or simply change position, and I realize how invisible these abilities are until you begin to lose them.

For me, movement is no longer something I can take for granted. Every step can carry a question: How many more steps will I have tomorrow?

The greatest fear I carry is not simply weakness. It is the possibility of losing my ability to walk one day because the treatment I need did not arrive in time.

And in muscular dystrophy, there is a phrase that carries enormous weight:

“Time is muscle.”

Why Is Time So Important?

My disease is progressive. This means that the longer the disease advances without an effective treatment, the more muscle function may be lost.

At the cellular level, repeated muscle injury and degeneration can eventually lead to the replacement of functional muscle tissue with fibrotic tissue and fat. Fibrosis means that connective tissue, particularly collagen, accumulates within muscle as part of an abnormal repair process. Over time, this reduces the amount of contractile muscle available to generate strength and can make restoring lost function increasingly difficult.

That is why time matters so much.

The goal is not simply to treat a number on a medical report. The goal is to preserve the muscles that allow me to stand, walk, breathe, move, and live independently.

Every month matters because muscle that is still functioning is muscle worth protecting.

The Hope of Gene Therapy

LGMD2D is caused by mutations affecting the SGCA gene, which encodes alpha-sarcoglycan, an important component of the muscle cell membrane. When this protein is deficient or absent, muscle fibers become more vulnerable to damage.

Gene therapy represents a fundamentally different approach: instead of treating only the consequences of muscle degeneration, it aims to address the underlying genetic cause by delivering a functional copy of the affected gene to muscle cells.

Modern gene-therapy research has demonstrated that this concept is not merely theoretical. Gene therapies for other rare genetic diseases have already produced remarkable scientific breakthroughs, showing that genetic medicine can transform diseases that were once considered extremely difficult or impossible to treat.

For someone living with a progressive genetic disease, these advances are more than scientific headlines.

They are hope.

They represent the possibility of preserving what I still have before more of it is taken away.

Why $2.3 Million?

A price of approximately $2.3 million for a gene therapy can sound impossible to understand at first.

But the cost of developing a treatment for an ultra-rare disease is fundamentally different from developing a medication for a condition affecting millions of people.

Gene therapies require highly specialized biological manufacturing, extensive research, rigorous quality control, clinical development, and complex delivery systems.

One important platform is the use of adeno-associated virus (AAV) vectors. These engineered viral vectors can be used as delivery vehicles to transport therapeutic genetic material into target cells. Manufacturing AAV-based therapies at the required clinical quality and scale is technically complex and expensive.

When a disease affects only a small number of people, the enormous costs of research, clinical trials, manufacturing, regulatory development, and long-term follow-up are distributed across a very small patient population.

The price reflects the extraordinary complexity and rarity of this field—but behind that number is something much more important to me:

the value of preserving a life that is still moving.

I Am Not Asking for More Time. I Am Asking for a Chance.

I cannot stop the clock.

I cannot change the mutation that I was born with.

And I cannot ask my muscles to wait patiently while science catches up.

But I can ask for a chance.

A chance to preserve my ability to walk.

A chance to keep the independence that remains.

A chance to wake up one morning without wondering whether yesterday was the last day I could do something as simple as walking across the room.

Scientific progress has already shown what is possible. Gene therapy has moved from an idea in laboratories toward real treatments for serious genetic diseases. That progress gives patients like me something we desperately need: a reason to believe that the future does not have to be defined entirely by decline.

My Greatest Fear—and My Greatest Hope

My greatest fear is that I will lose the ability to walk before treatment becomes accessible to me.

My greatest hope is that science moves quickly enough to change that story.

I do not want people to see only a rare disease, a genetic mutation, or a price tag.

I want them to see a person.

Someone who remembers what it was like to move without thinking.

Someone who still dreams, still hopes, still fights.

Someone whose future should not be determined by how quickly a progressive disease can destroy muscle.

Because for me, time is not just time anymore. Time is muscle. Time is movement. Time is independence. Time is life.

And that is why every day matters.

With Gratitude

To every scientist, physician, researcher, patient advocate, family member, donor, and person who believes that rare diseases deserve attention and hope: thank you.

Thank you for advancing science when the path is difficult.

Thank you for refusing to accept that rarity should mean invisibility.

And thank you for helping bring us closer to a future in which genetic diseases are not simply managed, but fundamentally changed.

I am grateful for every person who chooses to stand beside patients like me.

Because sometimes, hope is not about knowing exactly what tomorrow will bring.

Sometimes, hope is simply having enough time for science to catch up.

Thank you for listening, for caring, and for believing that my future is worth fighting

With love

Anwar ❤️