Our beautiful, bright 6 year old daughter Penny has recently been diagnosed with an ultra rare form of congenital muscular dystrophy called Selenon Related Myopathy SEPN-1. In spite of some challenges, Penny is currently relatively healthy and happy, however SEPN-1 is progressive, ultimately leading to respiratory failure, and worsening disability. Penny, like all children with SEPN-1, faces an uncertain future. Presently, no treatment is available.

A primary fundraising goal of Penny’s Promise is raising funds to seek treatment for SEPN-1. One key recipient of our fund will be the Beggs Laboratory and the Boston Children’s Hospital, where Dr. Alan Beggs is working on developing a treatment using gene therapy to treat SEPN-1. The Beggs Lab is one of the only places in the world that SEPN-1 is being studied. The disease is classified as ‘ultra rare’ (1 in 2,000,000) so funding for research into treatment is entirely inadequate.

By raising funds for researchers at Boston Children’s Hospital we will support the Beggs Lab to get the treatment ready for clinical trials sooner. Speeding up the timing of a treatment is so crucial for Penny. We are hopeful we can get her treatment before she has to contend with the worsening effects of the disease. Penny’s Promise will also be continuing to look to support efforts to develop new therapeutics that can improve the lifespan and quality of life with those living with neuromuscular conditions. All funds will be directed to this overarching goal. Penny loves life and has so much promise. It is our promise to her that by raising these funds, we will do everything we can to support her, and other children like her, have the best chance at a full and healthy life that we can.

 

Please note: Large donations are best made by cheque to avoid the 3.75% processing fee associated with online donations. If you wish to send a cheque, please make it out to “Toronto Foundation” and put “Penny’s Promise” in the memo section.

If you wish to make a gift of securities you can do so by filling out this form and following the instructions included on the form. Please reach out to Sarah Muir at smuir@torontofoundation.ca if you have any further questions.

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