Little Heroes – Video
Posted: February 2, 2013 at 9:41 pm
Little Heroes
Synopsis: Canavan Disease is a rare genetic brain disorder that is unknown to most non-neurologists. It starts at birth with severe psychomotor delays and seizures, and leads to premature death. Until recently, no disease-modifying therapy has been available. This is the true story of Dr. Paola Leone and her team as they encounter the young patients who led them to undertake the first gene therapy for a neurodegenerative disease. Leone was approached by families with no expectation of a cure, but driven by a "forward-dawning hope" that their participation would redefine what is possible in modern therapy. Canavan Disease represents a striking example of people coming together to make medical history, and their cooperative efforts in search of a cure continue to this day. This film is about the "Little Heroes" who spearheaded the viral-based gene therapy which is now being applied in clinical trials for Parkinson #39;s, Alzheimer #39;s, and other disorders.
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Little Heroes - Video
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