biotech / news / / The Irish Times
The baby girl was diagnosed with Canavan disease at birth.
An Irish child received experimental gene therapy for Canavan disease at Dayton Children’s Hospital in Ohio.
KEY POINTS
- Initial results show positive signs like reduced brain swelling and increased alertness within weeks of treatment.
- Prof Paola Leone’s team reported positive outcomes from eight children, including the Irish baby, in Nature Medicine.
- The biotech firm Myrtelle plans to seek FDA approval in 2024, with possible treatment approval by 2027.
- Earlier intervention with gene therapy appears critical, as older children may not benefit if brain damage has progressed.
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