Articles related to GENE THERAPY
COVID Vaccine Shedding Is ‘Real,’ FDA and Pfizer Documents Are Proof: Clinicians
Healthcare professionals are navigating a complex dialogue on COVID-19 vaccine shedding, underscored by FDA acknowledgments and Pfizer protocols that hint at the nuanced risks of gene therapy product exposure.
All Specialties February 22nd 2024
Gene Therapy Versus Common Care for Eligible Individuals with Sickle Cell Disease in the United States
The comparative modeling analysis of gene therapy for sickle cell disease underscores its potential cost-effectiveness and value in enhancing patient quality of life. With estimated ICERs significantly varying under different perspectives, the findings advocate for a nuanced approach to assessing novel therapies within the broader healthcare landscape.
Hematology February 7th 2024
Approved Roctavian ‘Liberating’ for 1st US Patient Treated Outside Trials
Roctavian represents a turning point in hemophilia A management, offering patients like Maxwell a potential lifetime of relief from traditional therapies. This gene therapy directly targets the genetic cause, paving the way for innovative treatments in genetic disorders.
Hematology January 22nd 2024
FDA Fast Track Designation and IND Clearance Granted to Novel Gene Therapy for GRN-Related Frontotemporal Dementia
The therapy aims to restore progranulin protein levels in the brain, marking a significant step forward in the treatment of neurodegenerative disorders.
Neurology November 14th 2023
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease
Learn how CRISPR-Cas9 gene editing holds the promise of elevating fetal hemoglobin levels and significantly reducing sickle cell disease manifestations. Explore the study’s findings to consider this potential game-changer for your practice.
Hematology September 5th 2023
Gene Therapy in Patients with the Crigler–Najjar Syndrome
This phase 1–2 study of gene therapy in Crigler–Najjar syndrome patients offers promising insights into a novel treatment approach. Explore the detailed findings to understand how this therapy could revolutionize the management of this rare and severe condition.
Gastroenterology August 22nd 2023