Family Raises $6M for FDA-Approved Treatment for Rare Disorder

A family in Natchitoches, Louisiana, is on a mission to raise $6 million to secure FDA approval for a treatment targeting a rare condition known as Sanfilippo syndrome, often referred to as “childhood dementia.” The urgency stems from the diagnosis of their daughter, three-year-old Lydia Rachal, who was identified with this condition after her mother, Morgan Rachal, recognized similarities with another child on TikTok.

Initially, Morgan Rachal, 30, believed her daughter was healthy, facing only minor issues such as ear infections and sleep disturbances. “When she was born in October 2022, the doctor said she was the perfect baby,” Morgan recalled, reflecting on their early days as a family. However, concerns began to mount when Lydia exhibited symptoms that did not align with typical childhood ailments.

While scrolling through TikTok, Lydia’s grandmother, Cindy Weaver, 54, came across a video of a girl who bore an uncanny resemblance to Lydia, including the same thick eyebrows—a characteristic associated with Sanfilippo syndrome. This prompted Morgan to consult their pediatrician, who agreed that Lydia’s appearance warranted further investigation.

After undergoing blood and urine tests, Lydia received a diagnosis of Sanfilippo syndrome B. This neurodegenerative disorder leads to a gradual loss of skills, earning it the nickname “childhood Alzheimer’s.” The diagnosis came with a stark prognosis; doctors informed the family that there is currently no cure and advised them to focus on creating memories with Lydia.

Morgan expressed the devastating impact of the news: “I felt like the life got sucked out of me. I felt like I couldn’t breathe.” The family learned that children with Sanfilippo syndrome often face severe developmental challenges and typically do not live past their twenties. As Morgan put it, “Her joy right now will be taken away if she doesn’t get into treatment.”

Refusing to accept defeat, Morgan, along with 14 other families affected by Sanfilippo syndrome, has identified an enzyme replacement therapy currently awaiting FDA approval. This treatment aims to provide the missing enzymes crucial for children with the disorder. According to the Cure Sanfilippo Foundation, while the therapy is still in clinical trials, it shows promise as a viable treatment option.

The families have already raised $1.6 million towards their $6 million goal, aiming to secure the necessary funds by spring 2026. Morgan emphasized the urgency, stating, “We need $3.8 million by December 1, 2025, and the rest by 2026.” Their hope is that by 2027, the treatment will be available for Lydia and other affected children.

“We are very hopeful that the treatment will be approved by the FDA,” Morgan stated. “This is our last chance to save Lydia. Currently, she doesn’t have any brain damage; she is your typical little toddler. My goal is to get her the treatment before the brain damage begins.”

The family’s journey underscores the power of community and shared experiences in the face of adversity. Morgan expressed gratitude for the support they have received, noting, “I am so grateful for everyone who is helping.” They have set up a GoFundMe page titled “Donate to Save Lydia” to facilitate donations and raise awareness about the condition.

As the Rachal family continues their efforts, they remain hopeful that their fight will lead to a breakthrough for Lydia and others battling this rare disorder.