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 ...
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 ...
Good News Network14h
Groundbreaking Gene Therapy Cures 21-year-old of His Sickle Cell Anemia: ‘I’m not in pain anymore’
Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...
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 ...
The American Journal of Managed Care6d
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 ...
Fierce Pharma14h
Bharat Biotech enters cell and gene therapy market with $75M production investment: reports
With a storied history in vaccine development, India's Bharat Biotech is shaking things up and answering the call of advanced ...
KXLY4d
Groundbreaking gene therapy research in Washington could lead to 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 ...
Preclinical study demonstrates improved gene therapy approach to treating a rare and devastating disease in children
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
Sarepta says teen died after its gene therapy treatment
Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy for a rare muscular dystrophy.
GEN - Genetic Engineering and Biotechnology News6d
DMD Patient Dies After Treatment with Sarepta Gene Therapy
The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...
University of California4d
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 ...