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Genetic Linkage

A Tribute to Max Randell, Gene Therapy Pioneer

I awoke on Monday morning to the sad news that Max Randell had passed away on April 18. He would have been 23 on October 9.

 

Maxie wasn't expected to live past the age of 8, or even much past toddlerhood, according to some doctors. But gene therapy, and his incredible family, had something to say about that. COVID-19 didn't claim him – his body just tired of fighting.

 

Max Randell's legacy is one of hope, to the rare disease community whose family members step up to participate in the clinical trials that lead to treatments. In this time of the pandemic, attention has, understandably, turned somewhat away from the many people who live with medical limitations all the time. I'll explore that story next week.

 

A Devastating Diagnosis

 

Max was diagnosed at 4 months of age with Canavan disease, an inherited neuromuscular disease that never touched his mind nor his ability to communicate with his eyes, even though his body increasingly limited what he could do. Fewer than a thousand people in the US have the condition.

 

To continue reading, please go to my DNA Science blog at Public Library of Science, where this post first appeared.

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