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Child Thriving After Becoming World's Youngest to Receive Gene Therapy for AADC Deficiency [Yahoo! Finance]

PTC Therapeutics, Inc. (PTCT) 
Last ptc therapeutics, inc. earnings: 4/30 04:01 pm Check Earnings Report
US:NASDAQ Investor Relations: ir.ptcbio.com
Company Research Source: Yahoo! Finance
CINCINNATI Nov. 15, 2024 /PRNewswire/ -- Sriansh Ojha was fully into his "terrible twos" on an April afternoon at Cincinnati Children's as he squealed and chased his older sister around the sunlit lobby of the Schubert Research Clinic. Urging the laughing little boy to slow down was a parenting task that his mother, Bhawana Dhakal , and father, Tirtha Raj Ojha , of Lexington, Ky. , felt profoundly pleased to perform. That's because in the first year of life, their son struggled to eat, could not lift his head off the bed, could not control his eye movements, and was not expected to ever be able to walk, much less run. "Gene therapy is a blessing I must say. You can see my child, before and after, and see that it works. He's a new person. He has a new life," Bhawana says. Sriansh was born with a rare genetic condition called AADC deficiency. In November 2022 , at age 16 months, Sriansh became the youngest child in the world to receive a remarkably successful one-time gene therap Show less Read more
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