Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells.
Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical ...
It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...
Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of ...
Market OverviewThe global cell and gene therapy market is undergoing rapid and transformative growth, reflecting its increasing importance in modern healthcare.The market is projected to expand at a ...
Clinical Trials Arena on MSN
Ultragenyx’s gene therapy shows sustained improvements in MPS IIIA
There are currently no approved disease-modifying therapies for MPS IIIA.
Ryuji Morizane, MD, Ph.D., of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, ...
Creative Biolabs is helping researchers overcome challenges of gene therapy research by providing analytical testing ...
A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a ...
New, well-characterized AAV reference standards enable accurate measurement of full and empty capsids, improving assay ...
Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...
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