It’s heart-wrenching to think that children in today’s world, in countries across the globe, are losing their lives far too early simply because they cannot access the medical treatments they so desperately need. This is the tragic reality for many children suffering from Duchenne Muscular Dystrophy (DMD), a devastating and rare genetic condition that leads to progressive muscle weakness and, ultimately, early death. The fact that life-saving treatments exist, yet these children are still dying due to a lack of access, is something that we cannot ignore. And the harsh truth is, we are all responsible for this crisis.
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Who is Guilty?
DMD Associations, Health Ministries and Drug Manufacturers are Guilty!
DMD is a genetic disorder that affects mostly boys and gradually robs them of their ability to walk, leads to respiratory and cardiac failure, and shortens life expectancy, often to the age of 20. However, there is hope. Treatments for DMD, such as exon skipping therapies and gene therapies, have been developed and approved by major health authorities like the FDA (U.S. Food and Drug Administration) and EMA (European Medicines Agency). These treatments offer children a chance at a better quality of life and even survival, if they are administered in time.
But despite these advancements, a heartbreaking and unjust reality persists: millions of children are still without access to these life-saving treatments. It’s a situation that should be unthinkable in the modern world, yet it is a daily battle for families around the globe. [Read More: Duchenne Muscular Dystrophy Market Grows: But Not All Families Have Access to Treatments]
Why Is This Happening?
The reasons behind this disparity are multifaceted and rooted in systemic issues. One of the primary barriers is cost. Advanced treatments like those for DMD are expensive, and in many low-income or middle-income countries, these treatments are simply out of reach for most families. In wealthier countries, where these treatments may be available, bureaucratic red tape, insurance coverage restrictions, and a lack of awareness and support in certain areas still result in children being denied access.
The Role of Health Ministries
We call on ministries of health worldwide to take immediate and meaningful action. These governments are responsible for ensuring the health and well-being of their citizens, including children who suffer from rare diseases like DMD. It is unacceptable that in 2025, children are still dying from treatable conditions. Health ministries must prioritize the availability and affordability of treatments for rare diseases, securing necessary funds and improving access to the best medical care.
Governments must also work to streamline approval processes for new treatments and ensure that families are not burdened with bureaucracy when they are fighting for their child’s life. Policies must be put in place to create a global network of support for these families, ensuring that no child with DMD is left behind because of where they were born or their family’s financial situation.
Manufacturers of DMD Treatments: Time for Action
Equally, pharmaceutical companies that manufacture DMD treatments have a moral responsibility. While they have made incredible strides in the development of therapies, their work cannot stop at the laboratory or the clinic. We need them to take responsibility for ensuring equitable access to these treatments worldwide. This means pricing treatments in a way that is affordable for all families, providing financial assistance programs, and collaborating with international health organizations to distribute treatments to underserved regions.
We understand that research and development require significant investments, but the profits generated from the treatments must not come at the expense of the lives of children who need them. If these companies are to truly claim that they are in the business of improving lives, their efforts must be directed at ensuring these life-saving therapies reach those who need them most.
Why We All Have a Responsibility
While ministries of health and manufacturers play pivotal roles, the rest of us are not exempt from responsibility. Every time we hear about children dying from DMD, we must not turn a blind eye. This is not just the responsibility of doctors, parents, or governments—it is a global issue that calls on the compassion and collective action of all of us. The injustice of children suffering from a condition that is treatable should not be allowed to persist simply because of geographical or financial boundaries. [Read More: The Challenge of Getting DMD Gene Therapy: Is Geography Destiny?]
Raising awareness, supporting relevant charities, and demanding accountability from pharmaceutical companies and governments are all ways in which we can make a difference. Each of us has the power to contribute to a world where no child dies unnecessarily from Duchenne Muscular Dystrophy. [Read More: Ask the DMD Associations in Your Country: When Will Duchenne Treatments Come to Our Country?]
A Call for Change
We must come together to demand that no child ever again faces death from a treatable condition like DMD simply because of the lack of access to necessary medical care. This is a fight for life, and it is a fight we cannot afford to lose. To the ministries of health, we urge you to take bold action. To the manufacturers of DMD treatments, we call on you to ensure your innovations reach every child in need. And to all of us, we ask for unwavering support in this global movement to save children’s lives.
The time for change is now. Let’s make sure that the next generation of children with DMD does not have to suffer or die due to our inaction. They deserve better—and so do we.
What Should Be Done?
- DMD Associations should stop dealing with pointless matters and bring together the Ministries of Health and pharmaceutical manufacturers.
- Manufacturers of DMD treatments must immediately apply for licensing in all countries.
- Cost of treatments need to be reduced to a level that every family can access. [Read More: Duchenne Muscular Dystrophy: Treatment & Cost]
A risk sharing model should be implemented as in Brazil
It is not normal for a one-time gene therapy, with an unknown duration of efficacy, to cost $3 million.
We want the Ministries of Health and pharmaceutical companies to come together and find a solution to this issue immediately. [Read More: In Brazil, the Government is Expected to Implement a Risk-Sharing Model with Roche for ELEVIDYS]
Individual Campaigns Are Not the Solution
Do you like seeing families organizing individual campaigns? Why are you still waiting to do something? What kind of conscience is this!
Individual campaigns are the last hope for families. Some will be successful and unfortunately some will not be able to raise the necessary money!
Go ahead and do something and be a hero!
We Call on Pharmaceutical Companies to Take Action
The primary concern of pharmaceutical companies is not the income they will earn at the end of the year, but rather their contact with the Ministry of Health to ensure that the disease of children with DMD slows down.
Share this article with the Health Ministry of your country, association you follow, the politicians you support, the DMD patients around you, and the pharmaceutical companies.
Together we are stronger.
