Dallas scientists granted $420K to test gene therapy for rare neurological disease
Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...
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 ...
Good News Network2d
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 ...
Science Daily5d
Gene Therapy News
Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...
Cell & Gene Therapies in Gastrointestinal Disorders Therapeutic Analysis Report 2024: Sales are Forecast to Reach $187 Million by 2030
The "Cell & Gene Therapies in Gastrointestinal Disorders: Therapeutic Analysis" report has been added to ResearchAndMarkets.com's offering.Both marketed therapies are prescribed when a patient has ...
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 ...
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 ...
The American Journal of Managed Care9h
Real-World Study Suggests DMD Gene Therapy Is Safe, Effective
Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort.
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 ...
University of California6d
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 ...
FOX 5 New York on MSN1d
Sickle cell patient declared disease free after groundbreaking therapy on Long Island
A sickle cell gene therapy treatment has allowed a Long Island man to turn a chapter in his life, with doctors declaring him ...
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 ...