Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I

Authors: Lalitha R. Belur, Avery K. Huber, Hillary Mantone, Mason Robertson, Miles C. Smith, Andrea D. Karlen, Kelley F. Kitto, Li Ou, Chester B. Whitley, Elizabeth Braunlin, Justin Furcich, Troy C. Lund, Davis Seelig, Carolyn A. Fairbanks, Nicholas Buss, Kwi Hye Kim, R. Scott McIvor

Source: Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 4, Pp 101369- (2024)

Subject Terms: MPS I, gene therapy, AAV9, intrathecal, intravenous, dose response, Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050124001852; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/03d89651f88d4d478f8e98a618f31356

Association of Acute Anti-inflammatory Treatment With Medium-term Outcomes for Coronary Artery Aneurysms in Kawasaki Disease

Authors: Kevin G. Friedman, MD, Brian W. McCrindle, MD, MPH, Kyle Runeckles, MSc, Nagib Dahdah, MD, Ashraf S. Harahsheh, MD, Michael Khoury, MD, Sean Lang, MD, Cedric Manlhiot, PhD, Adriana H. Tremoulet, MD, MAS, Geetha Raghuveer, MD, Elif Seda Selamet Tierney, MD, Pei-Ni Jone, MD, Jennifer S. Li, MD, MHS, Jacqueline R. Szmuszkovicz, MD, Kambiz Norozi, MD, PhD, Supriya S. Jain, MD, Angela T. Yetman, MD, Jane W. Newburger, MD, MPH, Carolyn A. Altman, MD, Brett R. Anderson, MD, MBA, MS, Mikayla Beckley, BS, Elizabeth Braunlin, MD, PhD, Jane C. Burns, MD, Michael R. Carr, MD, Nadine F. Choueiter, MD, Jessica H. Colyer, MD, MBA, Frederic Dallaire, MD, PhD, Sarah D. De Ferranti, MD, MPH, Laurent Desjardins, MD, Matthew D. Elias, MD, Anne Ferris, MBBS, Michael Gewitz, MD, Therese M. Giglia, MD, Steven C. Greenway, MD, Kevin C. Harris, MD, MHSc, Kevin D. Hill, MD, MSc, Michelle Hite, Thomas R. Kimball, MD, Shelby Kutty, MD, Lillian Lai, MD, MHA, Simon Lee, MD, Ming-Tai Lin, MD, PhD, Tisiana Low, MD, MSc, Andrew S. Mackie, MD, MSc, Wadi Mawad, MD, Mathew, MSc, Kimberly E. McHugh, MD, Tapas Mondal, MD, Kimberly Myers, MD, Michael A. Portman, MD, Claudia Renaud, MD, Rosie Scuccimarri, MD, S. Kristen Sexson Tejitel, MD, PhD, MPH, Karen M. Texter, MD, Deepika Thacker, MD, Sharon Wagner-Lees, RN-BC, BSN, Kenny Wong, MD, Mei-Hwan Wu, MD, PhD, Varsha Zadokar, MBBS

Source: CJC Pediatric and Congenital Heart Disease, Vol 1, Iss 4, Pp 174-183 (2022)

Subject Terms: Diseases of the circulatory (Cardiovascular) system, RC666-701

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2772812922000483; https://doaj.org/toc/2772-8129

Access URL: https://doaj.org/article/1a1d47ee96ea419f8fe01dc25ed1396f

Pediatric SubQ-ICD implantation, a single center review of the inter-muscular technique

Authors: Daniel Cortez, Kari Erickson, Gurumurthy Hiremath, Nathan Rodgers, Brenda Dugas, Elizabeth Braunlin, Rebecca Ameduri, Jamie L. Lohr

Source: Indian Pacing and Electrophysiology Journal, Vol 21, Iss 1, Pp 25-28 (2021)

Subject Terms: Subcutaneous ICD, Pediatrics, Intermuscular, Diseases of the circulatory (Cardiovascular) system, RC666-701

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S0972629220301467; https://doaj.org/toc/0972-6292

Access URL: https://doaj.org/article/7585efa99c6446b4b60a2e91dac41592

Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance

Authors: Mehmet Umut Akyol, Tord D. Alden, Hernan Amartino, Jane Ashworth, Kumar Belani, Kenneth I. Berger, Andrea Borgo, Elizabeth Braunlin, Yoshikatsu Eto, Jeffrey I. Gold, Andrea Jester, Simon A. Jones, Cengiz Karsli, William Mackenzie, Diane Ruschel Marinho, Andrew McFadyen, Jim McGill, John J. Mitchell, Joseph Muenzer, Torayuki Okuyama, Paul J. Orchard, Bob Stevens, Sophie Thomas, Robert Walker, Robert Wynn, Roberto Giugliani, Paul Harmatz, Christian Hendriksz, Maurizio Scarpa, MPS Consensus Programme Steering Committee, MPS Consensus Programme Co-Chairs

Source: Orphanet Journal of Rare Diseases, Vol 14, Iss 1, Pp 1-25 (2019)

Subject Terms: Morquio a syndrome, Mucopolysaccharidosis, MPS IVA, Management guidelines, Elosulfase alfa, VIMIZIM, Medicine

File Description: electronic resource

Relation: http://link.springer.com/article/10.1186/s13023-019-1074-9; https://doaj.org/toc/1750-1172

Access URL: https://doaj.org/article/07788539a8b74b2fa1444bee0867e560

Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance

Authors: Mehmet Umut Akyol, Tord D. Alden, Hernan Amartino, Jane Ashworth, Kumar Belani, Kenneth I. Berger, Andrea Borgo, Elizabeth Braunlin, Yoshikatsu Eto, Jeffrey I. Gold, Andrea Jester, Simon A. Jones, Cengiz Karsli, William Mackenzie, Diane Ruschel Marinho, Andrew McFadyen, Jim McGill, John J. Mitchell, Joseph Muenzer, Torayuki Okuyama, Paul J. Orchard, Bob Stevens, Sophie Thomas, Robert Walker, Robert Wynn, Roberto Giugliani, Paul Harmatz, Christian Hendriksz, Maurizio Scarpa, MPS Consensus Programme Steering Committee, MPS Consensus Programme Co-Chairs

Source: Orphanet Journal of Rare Diseases, Vol 14, Iss 1, Pp 1-21 (2019)

Subject Terms: Maroteaux-Lamy syndrome, Mucopolysaccharidosis, MPS VI, Management guidelines, Galsulfase, Enzyme replacement therapy, Medicine

File Description: electronic resource

Relation: http://link.springer.com/article/10.1186/s13023-019-1080-y; https://doaj.org/toc/1750-1172

Access URL: https://doaj.org/article/4b4cdd98c75840e4a9c116d9983d5417

A conserved HH-Gli1-Mycn network regulates heart regeneration from newt to human

Authors: Bhairab N. Singh, Naoko Koyano-Nakagawa, Wuming Gong, Ivan P. Moskowitz, Cyprian V. Weaver, Elizabeth Braunlin, Satyabrata Das, Jop H. van Berlo, Mary G. Garry, Daniel J. Garry

Source: Nature Communications, Vol 9, Iss 1, Pp 1-18 (2018)

Subject Terms: Science

File Description: electronic resource

Relation: https://doaj.org/toc/2041-1723

Access URL: https://doaj.org/article/e834b139d7d24de2a7e908969edba69b