Our son Samuel was diagnosed with Lysosomal Acid Lipase Deficiency (LAL-D) before he was born. LAL-D is a rare disease, believed to be significantly under-diagnosed that affects the body’s ability to break down fat and cholesterol, commonly leading to liver disease and high cholesterol. Sam is in good health because fortunately there is a treatment through enzyme replacement therapy. Sam receives bi-weekly infusions at our local Children's Hospital. He will most likely need to receive these treatments for the rest of his life.

We recently learned about Dr. Martin at Nationwide Children's Hospital. He has been developing a cure for LAL-D through gene therapy. Although this research is in its early stages, Dr. Martin says that initials experiments are showing promising results. Such treatment would be life changing for Sam, and many others who are affected by this disease.

Gene therapy is a new treatment and the path to FDA-approval is lengthy. Dr Martin will still need more research and resources before he can partner with established pharmaceuticals, and make this a reality.  We are creating this fundraiser to help pay for Dr. Martin's continued research and the costs related to this multi-year project.

Thank you for visiting our page and please consider donating or sharing. All donations are tax-deductible and will be acknowledged by Nationwide Children’s Foundation.