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Pharmaceutical Technology4h
Entering the era of prenatal gene therapies
As scientists explore treating genetic disorders before birth, the promise of foetal gene therapy is growing—but there are ...
Boy with ‘death sentence’ disease living a normal life after breakthrough gene therapy
A four-year-old boy with a life-threatening immune disease is now living a normal life thanks to a pioneering gene therapy ...
First patient to receive gene therapy treatment developed at Children's Hospital of Philadelphia living pain-free
The first patient to receive a new gene therapy at the Children's Hospital of Philadelphia is now living pain-free for the ...
Gene therapy delivered early can help children with rare neurodegenerative disease retain motor and cognitive functions
If administered early, gene therapy has the potential to change the medical history of children born with metachromatic ...
Investigational gene therapy gives children with rare immune disorder a new lease on life
An investigational gene therapy has successfully restored immune function in all nine children treated with the rare and life ...
In a first for St. Louis, patient completes new gene therapy to cure sickle cell disease
Martin Mwita, 20, is the first patient in St. Louis and among the first across the country to receive a new gene therapy for ...
SMA News Today2d
Zolgensma gene therapy liver damage usually resolves in months
Liver damage related to Zolgensma gene therapy usually resolves within a few months of treatment, a study found.
Muscular Dystrophy News10d
Sarepta: Gene therapy Elevidys has given ‘hope’ to DMD community
It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...
Breakthrough gene therapy gives boy with ‘death sentence’ disease a normal life
A boy with an ultra-rare immune disease which can carry a “death sentence” is living a normal life after taking part in a ...