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Rare Disease Day at NIH 2024 | National Center for Advancing Translational Sciences
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Expert Office Hours offered at RARE Drug Development Symposium
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This boy was born without an immune system Gene therapy rebuilt it
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TUBA CITY, Ariz. - Hataałii Tiisyatonii "HT" Begay, a Native American boy who is Diné, or Navajo, rolls toy trucks around in the dirt. He chases chickens into their coop. He pauses to watch his dad deftly gut a sheep for a ceremonial feast. Then he climbs onto a picnic table with his brother and cousins, chanting "Lava!" while pretending the ground has turned into molten rock. To his family, HT is a typical 5-year-old - a smiling, curious, busy blur who roams across his family's ranch, where the
This boy was born without an immune system Gene therapy rebuilt it
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