Therapeutic gene transfer to specific brain regions in animal models of Alzheimer's disease has uncovered multiple potential treatment approaches that deserve to be considered as candidates for ...
A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for ...
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Is your SCIg therapy for PI working? How to spot breakthrough infections
SCIg helps people with certain types of primary immunodeficiency reduce their infection risk. But that doesn’t mean you won’t ...
Although an inspiration for science fiction movies, gene therapy is real. Here is how it works and what could be improved ...
NEW YORK, February 26, 2026--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare ...
Orchestra BioMed has announced that the FDA has granted Breakthrough Device Designation for its atrioventricular interval modulation (AVIM) therapy, aimed at treating over 7.7 million U.S. patients ...
Expedited FDA pathways are boosting oncology and rare-disease drug development, with gene therapy, immunotherapy and precision medicine creating key opportunities.Dublin, Sept. (GLOBE NEWSWIRE) -- ...
Nipocalimab 15mg/kg improved ClinESSDAI score compared with placebo (treatment difference -2.65; [90% CI, -4.03, -1.28]; P =.002). The Food and Drug Administration (FDA) has granted Breakthrough ...
The FDA has granted Breakthrough Therapy designation to GLM101 for phosphomannomutase 2 congenital disorder of glycosylation.
High dose and low dose AMT-130 demonstrated an 80% and 30% slowing of disease progression vs external control, respectively. The Food and Drug Administration has granted Breakthrough Therapy ...
Blood specimen tubes are seen in the laboratory of Bellville Medical Center in the state of Texas where hospitals are overwhelmed due to record Covid-19 ...
The FDA will speed up their review of Enhertu for HR-positive, HER2-low/ultralow advanced breast cancer that cannot be removed via surgery. The Food and Drug Administration (FDA) granted a ...
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