As the son of two geneticists, science has always been a part of my family. I never entirely knew the nature of my father's research, except that he worked on a disease called Spinal Muscular Atrophy (SMA). This disease is a rare, genetic, neuromuscular disease which causes progressive muscle weakness. It affects 1 in every 10,000 births and is the number one cause of genetic death in infants. While working in my father’s lab, I became entrenched in the science of SMA and started to learn that the field was on the verge of a huge breakthrough. I knew I had to start filming the community and follow the events as it lead up to new discoveries in the field. Then I met Miles and his family.
Miles is a five year old boy with type II SMA. Like many SMA families, his parents, Nikki and Tony, were shocked by the diagnosis but fought to find an experimental treatment for their son. Following the family into a clinical trial, I saw how their story embodied the hope and struggle all SMA families go through when faced with the disease. Miles enrolled in a clinical trial and began taking an experimental drug to help treat his disease, which would later go on to be the first FDA approved drug for SMA. This disease has gone from an orphan disease with nearly nothing known about it, to now having its first FDA approved drug on the market, as well as other treatments in the pipeline. This incredible achievement represents the dedication of a community towards finding a treatment. The film represents what can be accomplished when families, researchers, and pharmaceutical companies join together to solve complex diseases.