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Barbara A. Konkle on Muck Rack

Barbara A. Konkle

As seen in: Color Research & Application, Nature, Wiley Online Library, The New England Journal of Medicine, The Lancet, Healio, HemOnc Today, Texas Medical Center
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Articles

The Critical Need to Consolidate All Gene Therapy Data in Haemophilia

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By Barbara A. Konkle, Donna Coffin, Cedric Hermans, Mayss Naccache
| Wiley Online Library Verified
Gene therapy for haemophilia has been in development for several decades, with the first clinical attempts initiated in the 1990s [1]. These early studies, including six initial trials, laid the foundation for subsequent systemic liver-directed adeno-associated virus (AAV) trials that ultimately led to regulatory approvals. A pivotal milestone came in 2011 with the publication by Nathwani et al.
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Comorbidities, Health-Related Quality of Life, Health-care Utilization in Older Persons with Hemophilia-Hematology Utilization Group Study Part VII (HUGS VII)

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By Randall Curtis, Marilyn J. Manco-Johnson, Barbara A. Konkle, Roshni Kulkarni
| Texas Medical Center
PURPOSE: We compare the impact of hemophilia on comorbidities, joint problems, health-related quality of life (HRQoL) and health-care utilization between two age groups: 40-49 years and ≥50 years. PATIENTS AND METHODS: The HUGS VII study recruited persons with hemophilia A or B age ≥40 years.
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BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

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By Barbara A. Konkle, Christopher Walsh, Miguel Escobar, Neil Josephson
| Texas Medical Center
Abstract Gene therapy has the potential to maintain therapeutic blood clotting factor IX (FIX) levels in patients with hemophilia B by delivering a functional human F9 gene into liver cells. This phase 1/2, open-label dose-escalation study investigated BAX 335 (AskBio009, AAV8.sc-TTR-FIXR338Lopt), an adeno-associated virus serotype 8 (AAV8)-based FIX Padua gene therapy, in patients with hemophilia B.
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