It’s a parent’s ultimate nightmare — witnessing your child’s health deteriorate right before your eyes. This is the heartbreaking reality for the family of Lily Bennett.
Lily suffers from a rare genetic disorder known as Sanfilippo Syndrome (MPSIII), a terminal and degenerative condition affecting roughly 1 in 70,000 births globally. Sadly, there is currently no cure or treatment available. However, promising gene therapy trials at Nationwide Children’s Hospital in Ohio have halted the progression of Sanfilippo in animal studies, with human trials tentatively slated for late 2014.
For children like Lily, the timing of these trials is crucial. Each passing day exacerbates the disease, bringing her closer to the inevitable life-altering and life-ending symptoms. Most children with this condition endure irreversible brain damage and typically lose the ability to communicate by the age of six. That’s a mere two years away for Lily. As her condition advances, she will gradually lose her mobility and the ability to care for herself, as seizures increasingly take a toll on her body. Her family is desperately holding on to the hope that these clinical trials will provide the answers and solutions they need.
Without sufficient financial support, the progress of these trials is in jeopardy, and time is slipping away for Lily and others battling this urgent disease. To delve deeper into Lily’s story, you can check out her Facebook page or follow her journey on Twitter @SavingLily #savinglily. You can also learn more about Sanfilippo Syndrome at the Cure Sanfilippo Foundation and explore ways to contribute to Lily’s fundraising efforts here.
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In summary, Lily’s family is fighting against the clock for a solution to her rare disease. They are hopeful that emerging clinical trials will bring much-needed treatments, but financial support is crucial to keep these trials moving forward.