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Ethan's Participation in a Clinical Trial results in a Treatment for DMD


Participating in a clinical trial results in a treatment for DMD

Sandra is a single mom raising her two boys. When her son Ethan was only 6 years old, he was diagnosed with Duchenne muscular dystrophy (DMD). DMD is a progressive muscle-wasting disease that robbed Evan of so many childhood joys – running, skipping, bike riding. At school, he could not participate in recess or gym as it caused too much muscle damage.  

DMD is a genetic disorder, but can also develop in families with no history of the disease.

Ethan's mobility gradually decreased, until at 12 years old he could no longer climb the stairs to the second floor, and Sandra had to install a chair lift.

Then Ethan was given the opportunity to participate in a clinical trial. He was so eager to participate, excited by the prospect of being able to help find a treatment or a cure for his friends.

However, the trial start date was delayed for four months, and by then Ethan couldn't even walk from the parking lot to the hospital treatment room.

When the trial finally started, Ethan received weekly infusions into a port in his chest, and every 12 weeks he had to travel to the Kennedy Krieger Institute in Baltimore for two days of testing, and also to Columbus, OH for deep tissue muscle biopsies.  

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Although the drug being tested in the trial (now called EXONDYS51) did not produce a cure, it was able to slow the progression of DMD deterioration. This is the very first treatment designed just for DMD patients and Ethan was thrilled to be part of a process that resulted in the drug becoming commercially available for himself and his friends.

After a few months on the experimental drug, Ethan went on a shopping trip to Target to help his older brother get supplies for his first year of college. As they left the store,  Sandra was amazed to realize they had just spent three hours walking the aisles and Ethan had not once complained of being tired.

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This content is for informational and educational purposes only. It is not intended to provide medical advice or to take the place of such advice or treatment from a personal physician. All readers/viewers of this content are advised to consult their doctors or qualified health professionals regarding specific health questions. CenTrial Data Ltd. does not take responsibility for possible health consequences of any person or persons reading or following the information in this educational content. Treatments and clinical trials mentioned may not be appropriate or available for all trial participants. Outcomes from treatments and clinical trials may vary from person to person. Consult with your doctor as to whether a clinical trial is a suitable option for your condition. Assistance from generative AI tools may have been used in writing this article.