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The Urgent Quest for a Cure: Eliza’s Battle Against Sanfilippo Syndrome
It is a harrowing reality for any parent to witness their child’s health decline. This is the heartbreaking situation faced by the family of young Eliza Thompson, who suffers from a rare genetic disorder known as Sanfilippo Syndrome (MPS III). This terminal and degenerative condition impacts approximately 1 in 70,000 births globally. As it stands, there is no known cure or effective treatment.
Significant progress has been made in gene therapy research at Nationwide Children’s Hospital in Ohio, where clinical trials have successfully halted the progression of Sanfilippo in animal models. Human trials are tentatively set to begin in late 2014, and for children like Eliza, the timing of these trials is critical. Each day that passes brings her closer to irreversible symptoms that could dramatically alter her life. Most children with Sanfilippo experience severe brain damage and lose the ability to communicate by the age of six. Unfortunately, for Eliza, that milestone is approaching rapidly.
As the condition advances, Eliza will face the loss of her mobility and ultimately the inability to care for herself, as seizures begin to take their toll. Her family clings to the hope that these upcoming clinical trials will deliver the answers and treatments they desperately seek. However, the continuation of these trials hinges on sufficient financial support, making it imperative to act quickly.
For those interested in learning more about Eliza’s journey, visit her story’s Facebook page or connect with her on Twitter at @SavingEliza #savingeliza. You can also read more about Sanfilippo Syndrome at the Cure Sanfilippo Foundation.
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In summary, the urgent need for funding and support for clinical trials to combat Sanfilippo Syndrome cannot be overstated. Every moment counts for families like Eliza’s, who are fighting against the clock for their children’s lives.