Baby given new gene therapy in groundbreaking trial
A baby boy with a rare illness is making “incredible” progress after he became the first ever patient to receive a new gene ...
University of California9h
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
STAT1d
Novartis finally delivers gene therapy data in older SMA patients. Will they still want it?
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
Gene therapy breakthrough may increase bladder cancer survival
But often the cancer will return and the entire bladder will be removed. “That comes like all kind of the side effects a ...
MedPage Today on MSN2d
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular Dystrophy
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
GEN - Genetic Engineering and Biotechnology News1d
Gene Therapy Tested in Mice Offers New Hope for People with Dravet Syndrome
By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...
KXLY10h
Groundbreaking gene therapy research in Washington could treat children living with severe epilepsy
Scientists at the Allen Institute have made a major breakthrough in research for people living with Dravet Syndrome, which is ...
Novartis builds case for new SMA gene therapy
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
Breakthrough gene therapy alleviates Dravet syndrome symptoms in mice without side effects
In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...