MedPage Today on MSN
Gene Therapy Restores Hearing for Rare Cause of Congenital Deafness
In a small pivotal trial of an investigational gene therapy, most cases of congenital profound deafness from genetic variants ...
A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...
Preclinical data demonstrates that Shape’s engineered AAV5 variant, SHP-DB1, efficiently targets the NHP brain after intravenous injection and ...
Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in ...
In February, the biotech reported improvements in 10 of 11 children who received DB-OTO. The AAV gene therapy, which ...
Regeneron's DB-OTO gene therapy shows hearing gains in nearly all CHORD study participants, with durable and meaningful improvements.
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...
Genethon, a worldwide pioneer and leader in research and development of gene therapy for rare genetic diseases, and Hansa Biopharma, a Sweden-based leader in ...
Researchers have achieved the first demonstration in mice of using gene therapy to reverse hallmark symptoms of SYNGAP1-related disorder, a devastating condition affecting an estimated 1 million ...
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