Publications by authors named "R E Gut"
Article Synopsis
- The study investigates the effects of gene therapy (etranacogene dezaparvovec) for treating moderate-to-severe hemophilia B, comparing it with traditional factor IX replacement therapy.
- A phase 3 open-label trial involved 54 men who received a single infusion of AAV5 vector after a lead-in period of factor IX prophylaxis, with the goal of reducing annualized bleeding rates.
- Results showed a significant decrease in bleeding rates from 4.19 to 1.51 and an increase in factor IX activity, demonstrating that gene therapy is not only noninferior but also superior to standard treatment.
Etranacogene dezaparvovec (AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized Padua variant human factor IX (FIX) transgene with a liver-specific promoter. Here, we report 3-year outcomes from a phase 2b, open-label, single-dose, single-arm, multicenter trial conducted among adults with severe or moderately severe hemophilia B (FIX ≤2%). All participants (n = 3) received a single intravenous dose (2 × 1013 gene copies per kg) and will be followed up for 5 years.
View Article and Find Full Text PDFEtranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including a gene cassette containing the factor IX (FIX) Padua variant under the control of a liver-specific promoter. A phase 2b study was conducted to confirm that a single dose of 2 × 1013 genome copies per kilogram of etranacogene dezaparvovec will result in FIX activity ≥5% 6 weeks after dosing. Secondary end points included FIX activity at other time points, bleed frequency, FIX replacement, and safety.
View Article and Find Full Text PDFHemorrhagic fever with renal syndrome (HFRS) is an acute viral zoonosis occurring due to the hantavirus infection. On the territory it constitutes the only case of viral hemorrhagic fever. The infections occurring in Poland are mainly caused by the Puumala and Dobrava serotypes.
View Article and Find Full Text PDFIntroduction: Standardized and disease-specific patient-reported outcome (PRO) instruments assessing pain, functional impairment and health-related quality of life (HRQoL) in people with haemophilia (PWH) have been used in studies, but infrequently in comprehensive care settings for individual assessment or treatment planning.
Aim: To assess the impact of pain and functional impairment on HRQoL in PWH.
Methods: P-FiQ enrolled 381 adult PWH with a history of joint pain/bleeding and included 5 PROs and a clinical joint evaluation (Hemophilia Joint Health Score v2.