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Jul. 9, 2018

TOPIC: Featured News, Patient Stories, Patients & Members, Uncategorized

Moving documentary on Joshua Frase Foundation, Nibs – the matriarch of the first ever MTM dog colony, and the power of patient advocacy

Posted by Laura Mullen

The Joshua Frase Foundation (JFF) is a non-profit organization founded with a simple yet seemingly unattainable vision: to find a cure for Myotubular Myopathy (MTM). The journey towards a cure started the day Joshua Frase was born with a disease so rare that he was said to be only one of fifty five known cases around the world. Not expected to live to see his first birthday, Joshua was sent home to die. His parents, Paul and Alison Frase, were told, “If he’s alive in a year bring him back and we’ll see what we can do.”  What the doctors didn’t know, and couldn’t predict, was that Joshua possessed an unquenchable desire to live.

 

By his first birthday, Paul and Alison decided to use Paul’s platform in the NFL and Alison’s relentless determination to save her son’s life, and jointly founded JFF. Over the next twenty two years, from a closet converted into an office, they raised over $7 million to fund cutting edge research. Gene therapy became the method of choice after a group in France, working with one of JFF’s researchers, found proof of concept with a small animal model. Working with JFF researchers, Alison took on the mission to find a large animal model in hopes of one day bringing this research to clinical trials.

 

The documentary linked below is the story of their journey taking an ultra-orphan rare disease to clinical trials. It is a story of using vision, fortitude, and resolve to push beyond perceived boundaries into new possibilities. This documentary is not just about the Frase family, but also about Nibs – the matriarch of the first ever MTM dog colony, and the power of patient advocacy. With it, the Frase family wishes to thank the incredible animal caretakers who provide excellent care for the dog colony at the University of Washington, the global team of researchers who have dedicated their tireless efforts, and to the families who have cared for these precious children, without them a cure would never be found.

 

Their journey to a cure began as a guarded hope to save their son’s life. Even though they were unable to accomplish that goal, today the hope is no longer guarded but fully engaged as they race towards a cure for Joshua’s peers.

 

The documentary is on the JFF website, please see link below;

 

https://www.joshuafrase.org/gamechanger-book.php