A Phase 3 Trial of l-Glutamine in Sickle Cell Disease von Niihara, Yutaka, Miller, Scott T, Kanter, Julie, Lanzkron, Sophie, Smith, Wally R, Hsu, Lewis L, Gordeuk, Victor R, Viswanathan, Kusum, Sarnaik, Sharada, Osunkwo, Ifeyinwa, Guillaume, Edouard, Sadanandan, Swayam, Sieger, Lance, Lasky, Joseph L, Panosyan, Eduard H, Blake, Osbourne A, New, Tamara N, Bellevue, Rita, Tran, Lan T, Razon, Rafael L, Stark, Charles W, Neumayr, Lynne D, Vichinsky, Elliott P

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A GH receptor antisense oligonucleotide inhibits hepatic GH receptor expression, IGF-I production and body weight gain in normal mice von Tachas, G, Lofthouse, S, Wraight, C J, Baker, B F, Sioufi, N B, Jarres, R A, Berdeja, A, Rao, A M, Kerr, L M, d’Aniello, E M, Waters, M J

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Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors von Mahlangu, Johnny, Oldenburg, Johannes, Paz-Priel, Ido, Negrier, Claude, Niggli, Markus, Mancuso, M. Elisa, Schmitt, Christophe, Jiménez-Yuste, Victor, Kempton, Christine, Dhalluin, Christophe, Callaghan, Michael U, Bujan, Willem, Shima, Midori, Adamkewicz, Joanne I, Asikanius, Elina, Levy, Gallia G, Kruse-Jarres, Rebecca

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Efficacy and safety of OBI-1, an antihaemophilic factor VIII (recombinant), porcine sequence, in subjects with acquired haemophilia A von Kruse-Jarres, R., St-Louis, J., Greist, A., Shapiro, A., Smith, H., Chowdary, P., Drebes, A., Gomperts, E., Bourgeois, C., Mo, M., Novack, A., Farin, H., Ewenstein, B.

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Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B von Pipe, Steven W., Leebeek, Frank W.G., Recht, Michael, Key, Nigel S., Castaman, Giancarlo, Miesbach, Wolfgang, Lattimore, Susan, Peerlinck, Kathelijne, Van der Valk, Paul, Coppens, Michiel, Kampmann, Peter, Meijer, Karina, O’Connell, Niamh, Pasi, K. John, Hart, Daniel P., Kazmi, Rashid, Astermark, Jan, Hermans, Cedric R.J.R., Klamroth, Robert, Lemons, Richard, Visweshwar, Nathan, von Drygalski, Annette, Young, Guy, Crary, Shelley E., Escobar, Miguel, Gomez, Esteban, Kruse-Jarres, Rebecca, Quon, Doris V., Symington, Emily, Wang, Michael, Wheeler, Allison P., Gut, Robert, Liu, Ying P., Dolmetsch, Ricardo E., Cooper, David L., Li, Yanyan, Goldstein, Brahm, Monahan, Paul E.

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Application of a hemophilia mortality framework to the Emicizumab Global Safety Database von Peyvandi, Flora, Mahlangu, Johnny N., Pipe, Steven W., Hay, Charles R. M., Pierce, Glenn F., Kuebler, Peter, Kruse‐Jarres, Rebecca, Shima, Midori

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US Guidelines for immune tolerance induction in patients with haemophilia a and inhibitors von Valentino, L. A., Kempton, C. L., Kruse-Jarres, R., Mathew, P., Meeks, S. L., Reiss, U. M.

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Inhibitors: our greatest challenge. Can we minimize the incidence? von Kruse-Jarres, R.

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Pocket handheld ultrasound for evaluation of the bleeding haemophilic joint: A novel and reliable way to recognize joint effusions von Zhou, J. Y., Rappazzo, K. C., Volland, L., Barnes, R. F. W., Brackman, M., Steiner, B., Kruse‐Jarres, R., Quon, D. V., Bailey, C., Chang, E. Y., Drygalski, A.

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The role of point-of-Care Musculoskeletal Ultrasound for Routine Joint evaluation and management in the Hemophilia Clinic - A Real World Experience von Gallastegui, N, Steiner, Buk, Aguero, P, Bailey, C, Kruse-Jarres, R, Quon, D V, Hanacek, C, Volland, L M, Barnes, Rfw, von Drygalski, A

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Establishment of a framework for assessing mortality in persons with congenital hemophilia A and its application to an adverse event reporting database von Pipe, Steven W., Kruse‐Jarres, Rebecca, Mahlangu, Johnny N., Pierce, Glenn F., Peyvandi, Flora, Kuebler, Peter, De Ford, Christian, Sanabria, Fabián, Ko, Richard H., Chang, Tiffany, Hay, Charles R. M.

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Alternative therapies for the management of inhibitors von Shima, M., Lillicrap, D., Kruse-Jarres, R.

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Presentation and management of acute coronary syndromes among adult persons with haemophilia: results of an international, retrospective, 10-year survey von Fogarty, P. F., Mancuso, M. E., Kasthuri, R., Bidlingmaier, C., Chitlur, M., Gomez, K., Holme, P. A., James, P., Kruse-Jarres, R., Mahlangu, J., Mingot-Castellano, M. E., Soni, A.

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Prevalence and Risk Factors for Hypertension in Hemophilia von von Drygalski, Annette, Kolaitis, Nicholas A, Bettencourt, Ricki, Bergstrom, Jaclyn, Kruse-Jarres, R, Quon, Doris V, Wassel, Christina, Li, Ming C, Waalen, Jill, Elias, Darlene J, Mosnier, Laurent O, Allison, Matthew

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Inhibitor recurrence after immune tolerance induction: a multicenter retrospective cohort study von Antun, A., Monahan, P. E., Manco‐Johnson, M. J., Callaghan, M. U., Kanin, M., Knoll, C., Carpenter, S. L., Davis, J. A., Guerrera, M. F., Kruse‐Jarres, R., Ragni, M. V., Witmer, C., McCracken, C. E., Kempton, C. L.

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Haemophilia A and inhibitors: advances in prevention and ITI treatment optimization von Kruse-Jarres, R.

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Positional Effect of Chemical Modifications on Short Interference RNA Activity in Mammalian Cells von Prakash, Thazha P, Allerson, Charles R, Dande, Prasad, Vickers, Timothy A, Sioufi, Namir, Jarres, Russell, Baker, Brenda F, Swayze, Eric E, Griffey, Richard H, Bhat, Balkrishen

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Prospective, observational study of plasma-derived factor VIII/von Willebrand factor in immune tolerance induction: the PRISM registry von Kruse-Jarres, R., Gilsenan, A., Spears, J., Kaye, J. A.

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Fully 2‘-Modified Oligonucleotide Duplexes with Improved in Vitro Potency and Stability Compared to Unmodified Small Interfering RNA von Allerson, Charles R, Sioufi, Namir, Jarres, Russell, Prakash, Thazha P, Naik, Nishant, Berdeja, Andres, Wanders, Lisa, Griffey, Richard H, Swayze, Eric E, Bhat, Balkrishen

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Unexpected pharmacokinetics of recombinant porcine factor VIII in a patient with acquired factor VIII deficiency and spontaneous epidural haematoma von Shatzel, J. J., Azar, S., Scherber, R., Gay, N., Deloughery, T. G., VanSandt, A., Kruse‐Jarres, R., Recht, M., Taylor, J. A.

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