Coming soon to a theater near you - the documentary Boys with Bigger Hearts (life with Hunter syndrome).
Filmmaker Joey Howell is currently developing a documentary about Hunter syndrome, or mucopolysaccharidosis II (MPS II). The initial inspiration for the film was near and dear to Mr. Howell’s heart. He has one cousin with MPS II and a few years ago another cousin, Tyson Cowan, passed away from the disease at the age of twenty-one. The film is truly a passion project with grassroots initiative. Using kickstarter to raise funds, Mr. Howell recently reached his goal of $30,000 to help finish the documentary and with 3 more days left in the fundraising, additional funds may enhance the film and its distribution. We at Rare Disease Report hope so.
MPS II is a lysosomal storage disease that mostly affects young males. Boys with MPS II are deficient in the enzyme iduronate-2-sulfatase that help breakdown glycosaminoglycans (GAG). The net result is that GAG builds up in cells and organs throughout the body and lead to a plethora of symptoms, including hearing loss, thickening of the heart valves, obstructive airway disease, sleep apnea, and enlargement of the liver and spleen. If not treated, boys with MPS II seldom live past puberty.
Mr. Howell wants to show the world what these boys’ lives are like. The film follows the lives of 4 patients with MPS II. A segment of the film can be seen at their boys with bigger hearts website and it shows the devastating impact this rare disease has on the patients as well as their families. The film also provides hope. Enzyme replacement therapy for MPS II is available (Elaprase®) and the film shows that such treatment can change these boys’ lives dramatically. Enzyme replacement therapy gives these boys a quality and quantity of life not possible a decade ago. However, the film also shows that treatment is very cumbersome and requires a strong commitment from the family to ensure it is administered properly.
The segment also reveals that these boys with bigger hearts not only have enlarged heart valves that limit their activity but they also have a passion and appreciation for life that is truly inspirational.
If you want to contribute to the film, please visit their fundraising website in the next few days. In an exclusive interview with Rare Disease Report, Mr. Howell said that he will use the funds to help complete filming over the next year as the boys continue to grow, undergo treatment, and/or get involved in new clinical trials. Mr. Howell is hopeful that in early 2014, the completed documentary will be available to help raise awareness of MPS II. Awareness that will allow these brave boys to lead more accepting lives in the community while waiting for better treatments and/or cures to be developed.