CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...
Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to ...
Neurogene has dropped plans to keep studying the high dose of its Rett syndrome gene therapy because of an adverse event that ...
In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...
In an update posted today, Neurogene said that the "emerging treatment-related serious adverse event (SAE)" was a case of ...
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...
Medpage Today on MSN4d
First Brain-Delivered Gene Therapy Approved for AADC DeficiencyThe treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...
Researchers have developed a dual-gene therapy approach to treat Usher syndrome type 1F, a rare condition causing deafness and progressive blindness.
Intellia presented data from its Phase I study of NTLA-2001 at the 2024 American Heart Association scientific meeting in ...
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
Wilson celebrates the first U.S. patient to complete gene therapy for sickle cell, but says advocacy must continue.
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
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