📢 Petition: Global Access to Life-Saving Treatments for Duchenne Muscular Dystrophy – Including Elevidys

 

We, the undersigned, call on governments, pharmaceutical companies, health organizations, and international regulatory bodies to take immediate and coordinated action to ensure equal and urgent access to all available treatments for Duchenne Muscular Dystrophy (DMD) — especially gene therapies such as Elevidys — regardless of a patient's country of residence or economic status.

 

Duchenne Muscular Dystrophy is a rare, progressive, and fatal neuromuscular disease that primarily affects young boys. It robs children of their mobility, independence, and eventually their lives. Time is critical. Every day without treatment leads to irreversible muscle loss and a decline in quality of life.

 

Elevidys, a gene therapy developed to target the root cause of DMD, offers hope for many families. Approved in the United States, it has shown the potential to slow or even halt the progression of this devastating condition. However, this groundbreaking treatment is not yet available in many countries — and where it is, it is often out of financial reach for most families.

 

This inequality is unjust.

 

We urge action on the following:

 

1. Global Approval Pathways – Fast-track Elevidys and similar therapies for approval and reimbursement worldwide.

 

 

2. Equity in Access – Establish compassionate access programs and international funding mechanisms to support children in countries without local availability.

 

 

3. Transparency – Demand that pharmaceutical companies disclose pricing models and make treatments accessible based on medical need, not nationality or wealth.

 

 

4. Research Support – Encourage continued development of treatments for all DMD mutations, including exon duplications and deletions, and ensure that children with rarer variants are not left behind.

 

 

5. International Collaboration – Create a global DMD task force to coordinate access, treatment guidelines, and patient registries.

 

 

 

Every child deserves a chance at life, no matter where they are born.

 

We stand united — parents, caregivers, doctors, advocates, and citizens — to demand action, compassion, and fairness.

 

Let’s bring hope home for every child with Duchenne.

Time is muscle. Time is life.

**I am Šárka, grandmother to William, a joyful and brave 4-year-old boy with Duchenne. William was misdiagnosed for almost 3 years. We were told it was autism — but it was Duchenne, silently damaging his muscles. Now, he has been accepted for gene therapy at Boston Children's Hospital, but the cost is overwhelming: $3.2 million for the infusion and over half a million for aftercare. We simply cannot raise this alone.

Time is against us, and William is losing strength. We will never give up. We are doing everything in our power to give him a chance. But we need the world to act — not just for William, but for every child still waiting.**

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