Ask the DMD Associations in Your Country: When Will Duchenne Treatments Come to Our Country?

Who is responsible for DMD treatments not coming to my country? Are DMD associations, scientists, and politicians making efforts to bring exon skipping or DMD gene therapies to your country? What factors determine whether they are successful?

We continue to voice the problems of DMD patients from many countries around the world. Duchenne muscle patients report that DMD associations in their countries are not working effectively. Meetings with politicians and pharmaceutical companies do not yield visible results. Who is responsible for DMD treatments not coming to my country?

What Are You Waiting For?

If FDA and EMA approved DMD treatments have not yet arrived in your country or are not covered by insurance, you should know that the DMD associations in your country are not doing enough to combat it. So, ask the DMD associations in your country: When will duchenne treatments come to our country?

Learn More: FDA-Approved Treatments for DMD

About DMD

Duchenne Muscular Dystrophy (DMD) is a rare and devastating genetic disorder that primarily affects boys, causing progressive muscle weakness, loss of mobility, and eventually leading to early death due to respiratory and cardiac complications. Over the past few decades, there has been significant progress in understanding the disease, and several treatments have been developed that can improve the quality of life for those affected.

Despite these advancements, DMD associations in the world have faced numerous challenges in ensuring that these life-changing treatments are accessible to patients in their respective countries. The gap between cutting-edge research, approved therapies, and patient access to those treatments continues to widen, and the inadequacy of DMD associations in bridging this gap cannot be ignored. [Read More: What is Duchenne?]

The Disparity in Treatment Access

One of the most glaring issues that DMD associations face is the disparity in access to approved treatments between countries. In high-income nations, such as the United States and many European countries, medications like eteplirsen, ataluren, and more recently, gene therapies are available to patients. These treatments, while expensive, offer hope for slowing disease progression and improving outcomes for affected individuals. However, in many low- and middle-income countries, access to these therapies remains either restricted or completely unavailable. The cost of these drugs, along with bureaucratic delays in approval processes, often prevents patients from accessing life-saving treatments in a timely manner.

In some cases, DMD associations have successfully lobbied for the approval and introduction of new treatments in certain countries. However, these efforts have often been isolated and lack the scale needed to make a significant impact globally. The inability to universally advocate for treatment accessibility or find viable solutions for lower-income countries is a major failure on the part of these organizations, whose primary goal should be to ensure that all children, regardless of their location or financial status, receive the care they deserve.

The Role of DMD Associations: Advocacy or Bureaucracy?

While DMD associations around the world play a crucial role in raising awareness about Duchenne Muscular Dystrophy, their advocacy efforts often fail to translate into tangible results for patients. In many cases, these organizations are bogged down by bureaucratic processes, struggling to navigate complex governmental and pharmaceutical landscapes to advocate for the rapid approval and distribution of new treatments. They may also be overly reliant on corporate partnerships, leaving them vulnerable to conflicts of interest that hinder their ability to act in the best interest of patients.

Additionally, many DMD associations fail to focus on or understand the needs of patients in low-income countries. Their efforts are focused on high-income countries where the infrastructure to deliver treatments already exists, which wastes time for children in other countries. This lack of a global perspective leads to a fragmented approach to DMD care that does not prioritize the most vulnerable populations.

DMD Associations’ Collaboration with Politicians

According to the information we received from DMD patients, DMD associations do not produce meaningful results from their meetings with politicians. It is not explained what actions will be taken as a result of the meetings and how they will be followed up. It is stated that the meetings are for show and do not go beyond giving the impression that they are working.

What Determines the Success of DMD Associations?

If treatments such as exon skipping and gene therapy have not yet arrived in your country or are not covered by insurance, then the work of DMD associations in your country is insufficient.

Meetings, photos taken and social media posts will not be enough to bring DMD treatments to your country.

If treatments have not yet arrived in your country, you should first make preasure the DMD associations in your country. You should never accept the excuses they make. Because the aim of the associations is not to make excuses, but to produce solutions.

Responsibilities of Doctors

If pediatric neurologists and geneticists in your country are not investigating the effectiveness of DMD treatments developed around the world or are not examining those receiving the treatments, you should know that there is a problem.

If scientists in your country are not doing conclusive studies on the genetics of DMD, but are constantly criticizing the treatments that are being developed and marketed, and are frequently saying that the treatments do not work, you should know there is a problem.

It is now very easy to reach children around the world who have received DMD exon skipping and gene therapies. We at DMD Warrior frequently communicate with the families of these children. Many of the families report that the treatments work wonderfully.

What Determines the Success of Doctors and Scientists?

If doctors and scientists in your country cannot make serious drug developments regarding DMD treatments, this means they are not successful.

If they don’t research treatments for children with DMD, don’t study the data, and don’t meet with families of children receiving the treatment, but are constantly criticizing current treatments, you should know they are not successful.

Responsibilities of Politicians

If politicians in your country think they will achieve success by taking a photo with a child with DMD and sharing it on social media, you should realize that this no longer works!

The main mission of politicians is to produce successful results, not excuses!

If all DMD treatments have not yet arrived in your country or are not covered by insurance, politicians are among those most responsible for this. [Read More: Duchenne Muscular Dystrophy: Treatment & Cost]

Responsibilities of Pharmaceutical Companies

If pharmaceutical companies have not yet applied for licensing for DMD treatments in your country, you must be wondering about the deeper reason behind this.

If pharmaceutical companies still do not set reasonable prices for the drugs they have developed to be distributed all over the world, all you need to know is that they are thinking about the money they will earn, not your children.

If associations, doctors, scientists and politicians are not making enough noise against these exorbitant prices, you should think carefully about the reason behind it.

The Need for Global Collaboration

One of the most significant obstacles in bringing Duchenne treatments to underserved regions is the fragmented nature of DMD advocacy. In order to achieve meaningful progress, a more unified, global approach is needed. International collaborations between DMD associations, pharmaceutical companies, governments, and non-governmental organizations (NGOs) could help expedite the delivery of treatments to countries that currently lack access to these therapies.

Organizations such as the World Health Organization (WHO) and Médecins Sans Frontières (MSF) have shown that large-scale, collaborative efforts can overcome barriers related to drug availability in low-resource settings. Similarly, DMD associations could learn from these models and adopt a more holistic and inclusive approach to global advocacy, focusing not only on wealthy nations but also on how to make treatments affordable and accessible for all.

Increasing the Pressure on DMD Associations

Ultimately, DMD associations must do more than raise awareness and fund research; they must take on a greater responsibility for pushing for universal access to care. This means stepping up their lobbying efforts not only with drug companies but also with governments and international regulatory bodies. It is not enough for these associations to simply ask for treatments to be made available in high-income countries; they must actively advocate for global solutions that address systemic barriers, such as the high cost of therapies, poor healthcare infrastructure, and limited funding for rare diseases in developing nations.

Increased pressure needs to be placed on these organizations to shift their focus towards global access, with a particular emphasis on regions that have been historically neglected. This will require strong leadership, transparency, and a renewed commitment to putting patients’ needs at the center of their efforts.

Conclusion

The inadequacy of DMD associations in your country ensuring that Duchenne treatments reach patients everywhere is a critical issue that needs to be addressed. While progress has been made in some countries, there is still a significant gap in care for many affected individuals, particularly in low- and middle-income nations. The path forward requires a more global, unified approach to advocacy, one that pushes for equal access to care and treatment for all Duchenne patients, regardless of their geographic location or economic status. Only then can we truly say that the fight against Duchenne Muscular Dystrophy is making progress on a global scale.

Share this article with the association you follow, the doctor you have your child examined with, the politicians you support, the DMD patients around you, and the pharmaceutical companies.

Together we are stronger.

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