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Baby KJ Muldoon, 1st to be treated with personalized gene therapy, released from hospital
Baby KJ was successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia and Penn Medicine in a historical medical breakthrough. Now after over 300 days in the hospital,
A novel gene therapy for Alzheimer's disease, which showed promise to protect the brain from damage and preserve cognitive ...
Unlike existing treatments for Alzheimer’s that target unhealthy protein deposits, the new approach developed at UC San Diego ...
Data show SPR301 lowered α-synuclein, a hallmark of Parkinson’s disease, in cellular models, indicating potential for efficacy at doses with favorable safety Demonstrated greater α-synuclein ...
In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...
The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...
Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...
A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.
RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...
As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...
New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...
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