Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
By Deena Beasley (Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing ...
Patient dies following muscular dystrophy gene therapy, Sarepta reports
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
Breakthrough for baby with rare illness in world-first gene therapy trial
Fourteen-month-old Tomas was diagnosed with ornithine transcarbamylase (OTC) deficiency when he was a few weeks old ...
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 ...
University of California17h
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 ...
Gene-Therapy Maker Sarepta Tanks After Patient Death
Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...
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 ...
Teen’s death following Sarepta DMD gene therapy underscores a risk seen for decades
The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...
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 ...
BioPharma Dive18h
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 ...
KXLY18h
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 ...
The American Journal of Managed Care2d
Bustling Gene Therapy Pipeline for Neuromuscular Diseases Brings Thorny Questions to the Clinic
The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...