It’s showtime for “Hemophilia the Musical,” a BioMarin-sponsored show created by teens. This past weekend, 25 teens with hemophilia came together in New York from around the country to write and direct the original six-song musical with a storyline around teens who are “different” because of medical conditions.
The one-hour show—to be performed on Monday at 1 p.m. and streamed on Facebook Live—follows high school students working through their challenges and learning how to embrace who they are and to follow their dreams. The themes and songs were inspired by the student performers’ own stories.
For BioMarin, which is developing a hemophilia gene therapy, it’s a way to introduce itself to the community in a uniquely creative and relevant sponsorship. Its hemophilia A treatment valoctocogene roxaparvovec, designated a breakthrough therapy by the FDA, is currently in phase 3 studies. BioMarin said recently that data expected early next year “could potentially allow submission of a marketing application for valoctocogene roxaparvovec through an accelerated approval pathway in the second half of 2019.”
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“There are lots of other players in this space, so we want to come in in a way that is appropriate and feels right for the community without talking about drugs in development," Debra Charlesworth, vice president of corporate communications at BioMarin, said. "This gives us an opportunity to share a little bit about who BioMarin is, what we stand for and how we approach rare disease."
The students who auditioned to participate in the musical spent the weekend before the show in New York attending a workshop with their families that included coaching and information sessions on relaxation and pain management as well as talks about the benefits and therapeutic value of the arts.
Jeremy Nobel, M.D., founder of the UnLonely Project, talked to the students and families about the physical effects of loneliness, a common problem for chronic disease patients, and the power of the arts to help make connections. Research shows that loneliness and isolation result in a 30% increase in risk for early death. Put another way, “Being lonely is like smoking 15 cigarettes a day,” Nobel said. His organization works to increase awareness of the toxicity of loneliness, reduce its stigma and create arts programs for people to help make social connections.
“Almost any major illness tends to be isolating for the people who have it and also to their caregivers,” he said. “It’s critical as part of the treatment to attend to the social health of people who have these disorders in the same way we attend to their mental and physical health.”
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Nobel will also do assessment testing for BioMarin to determine the aggregate health benefit of the show production and weekend together. He and his team will be looking at factors such as the teens’ sense of connection, improved sense of confidence in managing hemophilia and reduced stress levels, he said.
The show’s musical director, Paul Russell, has experience with the hemophilia community as the vocal coach and director of the U.K. Haemophilia Society choir. Hemophilia advocate and CEO of Believe Limited, Patrick Lynch, was the workshop director, while actor Sam Underwood, known for his roles on Dexter and Homeland, served as one of the teen’s coaches.