I die of fatal disease. Bureaucrats should not decide how doctors behave toward me

At the age of 6, my fatal disease was diagnosed. At the age of 11, I lost my ability to walk. Today I am 23 years old and fight for my life – and for all those who are desperate for the cure.

Tragically, federal government stands on the road.

I’m not alone; Tens of millions of Americans suffer from rare diseases. And while I believe we have entered the “golden age” of personalized Medicine Adapted DNA of the Patient, Road regulatory blockade often forces us to wait for years to approach treatments that could help. We have to break this logam in so that patients can access top treatments today.

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My illness is Duchenne muscular dystrophy (DMD) – a rare, genetic disorder that causes the waste of my muscles. Patients lose their ability to walk for about 9 to 12 years and become fully dependent on strength. Then they notice the function of hands in teenagers noticeably and eventually lose their lives in the mid-20s, as the affected muscles are responsible for breathing, as well as the heart muscle.

I decided early not to let this disease define me – or stop me. At the age of 15, I founded a non -profit organization called Unist Duchenne. I continued to become a bestseller author, a public speaker, a Hollywood writer and producer, a biotechnical advisor for five companies, an investor and acknowledged the key leader of the opinion (KOL) in DMD.

Still, I still know the brutality and the pain this disease causes. My younger brother, who is 17 years old, also has DMD. Like my second younger brother, who died of illness at the age of 14. Duchenne affects not only a person with the disease, but also on everyone around them, because he steals a person from those who love them.

Fortunately, I am optimistic about innovation in the medical field. As someone who works in the biotechnical industry, I know countless progress that are currently in the works that will change the way we deal with the disease. With the ascent of Ai, An even greater progress can be achieved faster and cheaper, and drugs can be even safer than their founding.

What is critical in rare genetic diseases is the time. It is almost always better to intervene earlier, because it is easier to preserve the existing function and to overcome some of the effects of the disease. For example, younger Duchenne patients have more muscle.

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They also have less fibrosis (tissue scar) and fat infiltration – two biological effects of the disease as time progressing, which further reduces mobility. It is tragic that some patients are at the end of their lives, where days can make a difference between life or dying.

On average, clinical trials for new treatments need about 10 years. Until then, the patient’s illness may have progressed outside the point where it can be meaningfully treated – or even died. We need legislation like “Right to try for individualized treatments. “It is an innovative reform that is now a law in six countries that puts power in the hands of a doctor and a patient to make these critical decisions when to try out new medicines that are adapted on the basis of genetics.

I can currently be willing to try a new treatment, a treatment manufacturer can be willing to allow me to try it, and the doctor may be willing to manage it – but my uncle is not willing and prevents me from receiving and treatment with rescue.

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This is completely backwards. After all, doctors know their patients better than a distant bureaucrat who does not even know that the patient exists. Why should I pray the federal government for permission to try to save my own life?

It is a reality for me that I will die, probably before I reach the Middle Ages. The average life span for people with Duchenne is 25 years, but it doesn’t have to be so.