Littleton Family Advocates for Rare Disease Treatment Amidst Hope and Concern
- A Littleton family in Colorado raises awareness for Spastic Paraplegia 50, a rare neurodegenerative disorder.
- Their children have been diagnosed with this condition, making it difficult for them to reach developmental milestones.
- The family is sharing their story amidst hope for treatment and uncertainty about the future.
The Lockard family from Littleton is grappling with the challenges of their children’s neurodegenerative condition, SPG 50, which hinders developmental milestones. Rebekah Lockard reflects on the early signs of the disease in her daughter Naomi, expressing the emotional toll of potentially losing a child to such a condition. The family’s journey took a hopeful turn when their son Jack participated in a clinical trial for a gene therapy treatment, developed by another parent facing similar challenges. On December 6, Jack received the experimental drug, leading to immediate improvements in his behavior and cognitive abilities. Rebekah noted that while Jack is still slightly behind his peers, he is making progress and not regressing further. This newfound hope, however, is tempered by concern for Naomi, who may not have access to the same treatment due to funding limitations for the clinical trial. Rebekah emphasizes the urgent need for financial support to continue the trial and ensure that children like Naomi can receive the necessary treatment. She advocates for increased awareness and funding for rare diseases, stressing the importance of allowing affected children to live pain-free and enjoy life. The Lockards remain committed to fighting for access to treatment, hoping to shine a light on the broader issues surrounding rare disease research and funding.