I’m starting this GoFundMe campaign in the United States, and all funds raised will be transferred directly to the clinic in Dubai, where doctors are already waiting for Artem to arrive for treatment.

Meet Artem Saprykin, he is an acquaintance of mine — he is not my son — but I cannot stand aside.

For years, I’ve been deeply involved in the rare disease community. I’ve helped raise support that contributed to saving dozens of children with rare diagnoses from Eastern Europe, and I cannot describe what it feels like each time a life is saved. It’s difficult to imagine how it can be otherwise.

Who Artem is — and what he’s facing

Artem is 5 years old, and he has Duchenne muscular dystrophy (DMD) — a severe, progressive genetic disease that gradually destroys muscle tissue and leads to loss of mobility and serious heart and lung complications over time.

His family’s path to diagnosis was long and painful. When Artem’s blood tests showed very high ALT, AST, and CK, doctors initially suspected hepatitis — but an infectious disease specialist confirmed he did not have it. Only later did the family learn that elevated ALT/AST/CK can be part of the DMD picture.

They went through countless doctors. The first genetic test (checking for deletions and duplications in the DMD gene) came back negative, giving them hope. But Artem’s symptoms persisted. Expanded testing for point mutations finally revealed the truth: a nonsense mutation in exon 44 of the DMD gene.

In July 2025, the family learned the diagnosis. From that moment, their life became one continuous fear — because DMD doesn’t pause.

Right now, Artem is still early in the course of the disease, but signs are already present: daily activities are becoming harder, he experiences pain and discomfort, and the disease is beginning to affect his posture and body structure (spine, legs, chest). Time matters.

Why gene therapy

For decades, there was no cure for Duchenne muscular dystrophy. Historically, DMD led to loss of walking ability in childhood and, for many boys, death in the teens or 20s.

Thanks to progress in cardiac and respiratory care, people with DMD are living longer today—but this is still a life-limiting disease, and families are still racing against time.

That’s why gene therapy matters: not as a perfect “cure,” but as a real chance to slow progression and protect quality of life while a child is still strong enough to benefit.

Until the 2000s, many boys with DMD did not survive much beyond their teen years — a reality that shows how urgently new treatments are needed.

We live in a time when scientific research is progressing rapidly. Scientists have learned how to tackle DMD at its root using gene therapy. It might not be a complete cure, but it is a real chance for improved quality of life.

The gene therapy for DMD, ELEVIDYS (delandistrogene moxeparvovec-rokl), uses a vector to deliver genetic instructions to the body so it can produce a functional form of dystrophin (often described as “micro-dystrophin”).

Where your donation goes

All funds go directly to the clinic in Dubai to cover Artem’s treatment. This fundraiser covers only a portion of the treatment costs. Please see the full treatment cost below.

How you can help

By supporting this campaign, you are helping save Artem, and his family will be forever grateful for this gift of life.

I strongly believe that gene therapy is the future. Your donation is a vote for science, progress, and hope for future generations of children with Duchenne.

With gratitude,

Zina Lara, an advocate for children with rare diseases.