One Child's hope: personal stem cells tested against rare muscle disease

NCT ID NCT05154851

Summary

This was a special access program for a single two-year-old child with a severe, rare genetic muscle disease. Doctors gave the child 14 infusions of his own specially processed stem cells over nearly a year to see if it was safe and if it could help control the disease. The study closely monitored the child's health and muscle function for over a year after the treatments ended.

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Contacts and locations

Locations

  • Hope Biosciences Stem Cell Research Foundation

    Sugar Land, Texas, 77478, United States

Conditions

Explore the condition pages connected to this study.