Delandistrogene moxeparvovec-rokl shows significant motor function improvements in 8- and 9-year-olds with DMD, countering expected motor decline. Statistically significant differences were observed ...
10d
First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy
Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...
Patients with Leber hereditary optic neuropathy (LHON) due to the MT-ND4 gene variant treated with the gene therapy lenadogene nolparvovec in one eye showed a sustained improvement in best-corrected ...
Please provide your email address to receive an email when new articles are posted on . Most patients who received Skysona for cerebral adrenoleukodystrophy did not have major functional disabilities ...
Ultragenyx Says Gene Therapy Shows Lasting Benefits, Slows Brain Decline In Fatal Childhood Disorder
MPS IIIA is caused by mutations in the SGSH gene, leading to a deficiency of the enzyme heparan N-sulfatase, causing toxic accumulation of heparan sulfate in the brain and body. It causes rapid ...
BOSTON (Reuters) - A year after receiving gene therapy for a condition that causes total blindness by age 30, three people continue to see better and one has improved enough to read the digital ...
After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...
Children with MLD treated with arsa-cel were less likely to develop severe motor impairment or die than typical among historical controls. Benefits of the gene therapy persisted over several years of ...
On Monday, Sarepta Therapeutics Inc. (NASDAQ:SRPT) released topline results from Part 2 of EMBARK (Study SRP-9001-301) Phase 3 clinical study of Elevidys (delandistrogene moxeparvovec-rokl), the only ...
Enrollment completion in the OCU400 Phase 3 clinical trial combined with positive 3-year data from the Phase 1/2 study are important accomplishments in the Company’s plan to begin bringing this ...
Gene Therapy Meets Regulatory Reality: The FDA–uniQure Clash Over a Promising Huntington’s Treatment
The FDA’s dispute with biotech firm uniQure over Huntington’s gene therapy highlights growing tensions between innovation, ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results