treatment

All that stands in the way of more children with SPG50 being treated is funding.

The gene therapy treatment “Melpida” is the only glimmer of hope that children with SPG50 and their families have. Below is a timeline showing children with SPG50 who have received the treatment.

Doses of Melpida are ready and waiting in refrigerators. All that stands in the way of more children with SPG50 receiving them is funding.

  • Michael Pirovolakis is diagnosed with SPG50. Michael’s dad Terry and mom Georgia set out to create a cure for the disease.

  • Michael becomes the first child treated with Melpida at SickKids in Toronto, Canada.

  • Jack Dwyer becomes the first patient treated in the United States after receiving Melpida at UT Southestern Medical Center in Dallas, TX.

  • Alberto becomes the third patient treated with Melpida. Alberto received treatment at UT Southwestern Medical Center in Dallas, TX.

  • Jack Lockard is treated with Melpida at UT Southwestern Medical Center in Dallas, TX. Jack becomes the youngest child ever to receive intrathecal (spine) gene therapy at 5 months old.

  • Alejandro becomes the fifth child treated with Melpida and the first child treated in Europe. Alejandro was treated at Biodonostia Health Research Institute in Spain.

  • Sergio becomes the sixth child to receive Melpida. Sergio was treated at Biodonostia Health Research Institute in Spain.

  • Ona is treated at one and a half years old at UT Southwestern. Another incredible success story getting a young child treatment before significant symptoms set in.

  • At 21 months Thomaz becomes the eighth child treated with Melpida at UT Southwestern

  • A Phase III Clinical trial has been approved for UT Southwestern, the final stage before the drug is approved by the FDA and other regulatory agencies. After approval, insurance and governments cover the cost of treatment for children, making it accessible to all.