The journey of the Rivera family has been one filled with challenges as they confront Sanfilippo Syndrome, often referred to as Childhood Alzheimer’s. After securing a groundbreaking clinical trial for their beloved daughter, Mia, the family is now dedicated to ensuring that other children facing similar battles can also access this life-altering gene therapy. “There are far too many children in need of help, and we can’t let them suffer while opportunities slip away,” stated Mia’s father, Daniel Rivera, in a heartfelt conversation.
Two years ago, the Riveras watched in despair as their 4-year-old daughter’s cognitive abilities began to decline. In their search for hope, they discovered a clinical trial at a leading medical facility in Columbus, Ohio. This prompted the family to embark on an ambitious fundraising campaign. Through the tireless efforts of their community, both online and offline, they managed to raise approximately $250,000—a commendable feat, but still not enough to cover the costs. Fortunately, renowned filmmaker Alex Thompson joined the cause, creating a viral video that ultimately attracted $2 million in donations for the trial.
Having raised sufficient funds for Mia and two other children, the Rivera family took an extraordinary step: they isolated themselves for 726 days. “We made the difficult choice to shield Mia from a specific virus that could have disqualified her from the trial. It was an arduous journey, but we persevered,” Daniel explained. With Daniel working from home, the family was able to commit to complete isolation. Mia’s mother, Lisa, left her job, and their older son, Jake, transitioned to online schooling. They implemented strict sanitization measures for everything that entered their home and relied on close friends and family for errands. “When we considered the criteria that could exclude Mia from her only chance at life, we knew isolation was necessary. We never imagined it would last that long,” Daniel shared.
Fast forward to May, when Mia became the first child globally to receive the innovative gene therapy for Sanfilippo Syndrome. “We’ve noticed a renewed spark in her eyes and a stronger connection with us—something we thought was lost forever. She’s much more involved in playtime activities now. Just the other day, she picked up a toy and brought it to us to play with together, which hadn’t happened in ages,” Daniel proudly recounted. Although Mia had lost several skills due to the disease, including her speech, there are now glimmers of improvement following the therapy. “You can see the desire to learn in her. She’s engaged, she’s trying, and she’s doing it all with a big smile!”
Since this breakthrough, the Rivera family has redoubled their efforts to raise funds and awareness for other children battling Sanfilippo Syndrome. Daniel is optimistic that gene therapy could be a game-changer for countless kids. “We’re extremely hopeful. Early signs suggest that the therapy is indeed showing positive effects,” he stated. With Alex Thompson’s assistance, they have produced another video aimed at increasing visibility and support for additional clinical trials. “This is just the beginning; it opens doors for every child in need, depending on the outcomes,” Daniel elaborated. “Every child deserves a fighting chance, don’t you think?”
Their mission now centers on gathering awareness and resources to assist families nationwide who have children with Sanfilippo Syndrome. “Mia’s legacy is about helping her friends receive the same opportunity she did. Miracles can happen for these little ones, but the time to act is now,” her father concluded.
For more insights into the journey of families facing similar challenges, check out our blog post here. And if you’re considering at-home insemination, visit Make A Mom for quality syringe kits. Additionally, for anyone interested in pregnancy resources, the NICHD offers invaluable information.
In summary, the Rivera family’s story highlights the power of community support and determination in the face of daunting medical challenges. Their commitment to helping others ensures that Mia’s journey becomes a beacon of hope for many.
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