Right-to-try Laws Give Last Minute Hope for Spinal Muscular Atrophy

Shortly after celebrating baby Eli’s birth, his family got the shocking news that Eli had a fatal disease called spinal muscular atrophy (SMA).

Spinal muscular atrophy can take away Eli’s ability to breathe and eat. This is because SMA damages and affects the motor nerve cells in his spinal cord. His family was told that he would not live to the age of one. Amazingly, he has made it to the age of two.

This is due to Eli being selected as a participant in a clinical trial. He was one of 15 kids who had access to an investigational treatment. Even though Eli received only a partial dose, at this point, he can talk and even sing. In addition, he can walk with the help of his walker.

Those that received a full dose of the treatment are no longer showing any symptoms related to the disease.

Eli’s situation is one of the few lucky ones. Many terminally ill patients do not have the time to wait for FDA approval of the promising drugs they need to live. A federal version of a right-to-try law is necessary because only 33 states have a similar law.

This law would allow those who are terminally ill and have exhausted treatment options to seek further treatment. This would allow them to at least try treatments that have already passed Phase 1 FDA Trials and are still trying to get the complete FDA approval.

An oncologist in Texas has treated 78 of his patients under that kind of law in his state. With these patients, he says that some are surpassing the projected life expectancy and most of the patients are doing well with the treatments. This offers hope to many other patients and their families.

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