Last Updated: 09/26/2026
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Found 13 publications
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.
Journal: The New England journal of medicine
Published: October 09, 2024
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.
Journal: Lancet (London, England)
Published: April 23, 2024
Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia.
Journal: Molecular therapy. Methods & clinical development
Published: March 16, 2023
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop.
Journal: Human gene therapy
Published: February 16, 2023
Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease.
Journal: The New England journal of medicine
Published: December 13, 2021
Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.
Journal: The New England journal of medicine
Published: December 10, 2021
A First-in-Human Study To Assess the Safety and Pharmacokinetics of LYS228, a Novel Intravenous Monobactam Antibiotic in Healthy Volunteers.
Journal: Antimicrobial agents and chemotherapy
Published: December 12, 2018
A first-in-human, randomized, double-blind, placebo-controlled, single and multiple ascending oral dose study to assess the safety, tolerability, and pharmacokinetics of BZF961 with and without ritonavir in healthy adult volunteers.
Journal: Journal of drug assessment
Published: June 15, 2018
The Safety and Antiviral Activity of BZF961 with or without Ritonavir in Patients Infected with Hepatitis C Virus: A Randomized, Multicenter Trial.
Journal: Clinical therapeutics
Published: May 24, 2018
Hepatitis B virus outreach to immigrant population in Greater Boston Area: Key to improving hepatitis B knowledge.
Journal: World journal of gastroenterology
Published: July 07, 2017
A Diacylglycerol Transferase 1 Inhibitor Is a Potent Hepatitis C Antiviral in Vitro but Not in Patients in a Randomized Clinical Trial.
Journal: ACS infectious diseases
Published: October 30, 2016
Preclinical Profile and Clinical Efficacy of a Novel Hepatitis C Virus NS5A Inhibitor, EDP-239.
Journal: Antimicrobial agents and chemotherapy
Published: April 19, 2016
Last Updated: 09/26/2026