Advent of oral medications for the treatment of hereditary angioedema

Autor: Anna Valerieva, Teresa Caballero, Markus Magerl, Joao P. Frade, Paul K. Audhya, Timothy Craig

Publikováno v: Clinical and Translational Allergy, Vol 14, Iss 9, Pp n/a-n/a (2024)

Abstract Background Hereditary angioedema (HAE) is a rare genetic disorder characterized by unpredictable, debilitating episodes of submucosal and/or subcutaneous tissue swelling, which may be life‐threatening depending on anatomic location. The tw

Externí odkaz: https://doaj.org/article/16eb12a496914dd2981f2fdef6712048

Hereditary Angioedema With Normal C1 Inhibitor: US Survey of Prevalence and Provider Practice Patterns

Autor: Riedl, Marc A., Danese, Mark, Danese, Sherry, Ulloa, Julie, Maetzel, Andreas, Audhya, Paul K.

Publikováno v: In The Journal of Allergy and Clinical Immunology: In Practice August 2023 11(8):2450-2456

Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L

Autor: Ivana F. Audhya, Shelagh M. Szabo, Andrea Bever, Fiona O’Sullivan, Daniel C. Malone, David Feeny, Peter Neumann, Susan T. Iannaccone, P. Jayasinghe, Katherine L. Gooch

Publikováno v: Journal of Patient-Reported Outcomes, Vol 7, Iss 1, Pp 1-12 (2023)

Abstract Background The progression of Duchenne muscular dystrophy (DMD) is characterized by loss of ambulation, respiratory insufficiency, cardiomyopathy, and early mortality. DMD profoundly impacts health-related quality-of-life (HRQoL). However, f

Externí odkaz: https://doaj.org/article/56e8e0813bd14337a96c803b7f7fba68

Psychometric evaluation of the PROMIS parent proxy mobility item bank for use in Duchenne muscular dystrophy.

Autor: Lowes LP; Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Le Reun CM; Biostatistics, Sainte-Anne, Guadeloupe, France., Alfano LN; Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Reash NF; Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Iammarino MA; Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Patel S; Patient-Centered Outcomes, Sarepta Therapeutics, Inc., Cambridge, MA, USA., Audhya IF; Patient-Centered Outcomes, Sarepta Therapeutics, Inc., Cambridge, MA, USA.

Publikováno v: Developmental medicine and child neurology [Dev Med Child Neurol] 2024 Dec 19. Date of Electronic Publication: 2024 Dec 19.

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