Right-To-Try Laws Help Baby With SMA in Minnesota

According to a report on postbulletin.com, there are 33 states that have right-to-try laws, giving people with life-threatening illnesses access to drugs that have not yet been approved by the FDA.

MORE: West Virginia boy hopes to get Spinraza treatment for SMA.

So long as the drug has safely completed the Phase 1 stage of clinical testing on humans, terminally ill people can apply to take the drug rather than wait for up to 10 years for it to get the FDA’s approval.

One of the patients who benefitted from Minnesota’s right-to-try laws is Eli. Diagnosed shortly after birth with type 1 spinal muscular atrophy (SMA), Eli’s family was told that it was unlikely that he would live beyond his first birthday. However, thanks to the right-to-try laws in the state, Eli was able to join a clinical trial with 15 other SMA patients for a new drug.

Eli is now two years old and doing much more than doctors believed he would. He can talk and sing, and even walk with the aid of a walker—milestones the family was not expecting to witness.

Although taking medication that’s still in the research phase offers no guarantees, being allowed to take these drugs offers hope for patients who may have exhausted all their other options.

MORE: Two families in Northern Ireland win the right for Spinraza treatment for their children with SMA.

SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

One comment

  1. Alexandra says:

    It’s wonderful that patients like Eli have obtained access to a medication that allow him the chance to to live a full life. As a rare disease patient advocate, I know how crucial it is for children to begin a treatment as soon as possible. However, Right to Try laws don’t give patients access to clinical trials – they aim to help patients that don’t qualify for clinical trials get access to experimental drugs. In fact, patients seeking drug under RTT can’t have been granted access to a clinical trial. In this case, it means that Eli did not receive his medication through a Right to Try law, and using his story to promote the cause is not doing right by those patients most in need.

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