Our beautiful, bright 5 year old daughter Penny has recently been diagnosed with an ultra rare form of congenital muscular dystrophy called selenon related myopathy SEPN-1. This disease is progressive, and currently there is no treatment available. We're looking to raise funds for the Beggs laboratory at Boston children's hospital, where Dr Alan Beggs is working on developing a treatment using gene therapy to treat SEPN-1. As the disease is considered 'ultra rare' funding for research into treatment for this disorder is totally inadequate. By raising $100,000 we can support the hiring of another researcher in the lab who can help get this gene therapy to clinical trial. We're hopeful we can support this becoming available as soon as possible, before Penny has to contend with the worsening effects of the disease.