Every parent’s deepest fear is to witness their child suffer, and this is the heartbreaking reality for the family of young Mia Thompson. Mia is battling a rare and terminal condition known as Sanfilippo Syndrome (MPS III), a degenerative genetic disorder affecting roughly 1 in 70,000 births globally. Sadly, there is currently no cure or effective treatment available. However, promising gene therapy clinical trials at Nationwide Children’s Hospital in Ohio have shown success in halting the disease in animal models and are preparing to initiate human trials, tentatively set for late 2014.
The Urgency of Clinical Trials
For children like Mia, the urgency of these trials is paramount. Each day that passes brings her closer to the debilitating symptoms associated with this cruel disease. Most children afflicted with Sanfilippo Syndrome experience irreversible brain damage and typically lose their ability to communicate by age six—an alarming reality for Mia, who has less than two years left before reaching this critical milestone. As the disease progresses, she will gradually lose her ability to walk and ultimately be unable to care for herself as seizures take their toll.
Hope and Financial Support
Mia’s family clings to the hope that these clinical trials could provide the necessary treatment to change their lives. However, without adequate financial support, the continuation of these trials is jeopardized, and time is of the essence for Mia and countless others facing this dire situation. You can learn more about Mia’s journey here, visit her story on Facebook, and connect with her on Twitter at @SavingMia #savingmia. For more information about Sanfilippo Disease, the Cure Sanfilippo Foundation is an excellent resource, and you can also explore Mia’s fundraising efforts here.
Additional Resources
If you’re considering family planning options, check out our post on fertility boosters for men at Make a Mom. Additionally, for authoritative insights on home insemination, visit Intracervical Insemination. For comprehensive guidance on treating infertility, the ACOG offers valuable information.
Conclusion
In summary, Mia Thompson’s battle against Sanfilippo Syndrome highlights the urgent need for clinical trials and funding to provide hope for families facing such devastating diagnoses. The support of the community can make a significant difference in advancing research and finding a cure.