Amber, 8, has GAN, a rare degenerative neurological disease. Friends of Patients at the NIH, helped pay the mortgage of Amber’s family to relieve financial stress so both parents could be with her at the NIH while she was in an experimental study. Amber’s medical team at the NIH is attempting to not only to arrest the disease, but repair the damage.

They are doing this by placing a good gene into a “virus envelope” and injecting it into the spinal fluid. The hope is that it will float throughout the nervous system and replace the damaged gene where needed. Her medical team and her parents are optimistic that the treatment is going well.

Friends of Patient at the NIH provided support to 17-year-old Dixon and his family last year when he came to the NIH seeking a stem cell transplant for Sickle Cell Disease.

Dr. John Tisdale, his physician, who has pioneered this experimental treatment, says it is safe to say Dixon is cured! What great words for Dixon’s family to hear!

Initially, Dixon’s father, Leonard, took him and his older sister, Flavia, to India from their native country of Uganda. Both children had Sickle Cell Disease.

The family could only afford treatment for one child at a time, so Flavia told her little brother, “You go first.” Unfortunately, Flavia died waiting for medicine.

When Dixon’s father heard about this treatment in the United States, he resolved to do everything possible to save Dixon’s life. Hear Dixon’s story in his own words, meet his father and the physician/researcher, Dr. John Tisdale.

Please help us help provide emergency financial support to many more NIH patients in clinical trials, like Dixon.