A mutation in TGFB3 associated with a syndrome of low muscle mass, growth retardation, distal arthrogryposis and clinical features overlapping with marfan and loeys-dietz syndrome von Rienhoff Jr, Hugh Young, Yeo, Chang-Yeol, Morissette, Rachel, Khrebtukova, Irina, Melnick, Jonathan, Luo, Shujun, Leng, Nan, Kim, Yeon-Jin, Schroth, Gary, Westwick, John, Vogel, Hannes, McDonnell, Nazli, Hall, Judith G., Whitman, Malcolm

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Cardiac R2 values are independent of the image analysis approach employed von Meloni, Antonella, Rienhoff Jr, Hugh Young, Jones, Amber, Pepe, Alessia, Lombardi, Massimo, Wood, John C.

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Response to "De novo mutation of the TGFB3 latency-associated peptide domain in a patient with overgrowth and Loeys-Dietz syndrome features" von Rienhoff Jr, Hugh Young

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A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload von Hugh Young Rienhoff, Jr, Vip Viprakasit, Lay Tay, Paul Harmatz, Elliott Vichinsky, Deborah Chirnomas, Janet L. Kwiatkowski, Amy Tapper, William Kramer, John B. Porter, Ellis J. Neufeld

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Phase 1/2a Trial of Bomedemstat with or without All- Trans Retinoic Acid (ATRA) in Advanced Myeloid Malignancies von Ross, David M., Stevenson, William S., Shortt, Jake, Cooney, Julian, Lewis, Ian, Rienhoff Jr, Hugh Young

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MSC Reprogramming and Aberrant Inflammatory Signaling in Myelofibrosis: Insights from Single-Cell RNA-Sequencing of Trephine Bone Marrow Cells von Basma, Naseer J, Strickson, Alexia J, Gurashi, Kristian, Kleppe, Maria, Rienhoff Jr, Hugh Young, Somervaille, Tim CP

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Mutational Analysis of the Genome Stability Factor BOD1L1 in Myeloproliferative Neoplasm von Higgs, Martin R, Browne, Sofia, Collinson, Ryan, Fuller, Kathy, Erber, Wendy N., Kleppe, Maria, Sirnosian, Ben, Natsoulis, Georges, Guo, Belinda, Rienhoff Jr, Hugh Young

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LSD1 Inhibition Enhances Antigen Presentation and Co-Stimulation in AML to Promote T Cell-Mediated Anti-Leukemia Immune Responses von Yan, Yu, Singh, Kanwaldeep, Hartung, Emily, Lee, Brendan, Prabagaran, Pradhariny, Khalaf, Dina, Garcia-Horton, Alejandro, Leber, Brian, Walker, Irwin, Lepic, Kylie L., Kleppe, Maria, Rienhoff Jr, Hugh Young Young, Bramson, Jonathan, Berg, Tobias

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Phase 2 Study to Assess the Safety and Efficacy of Bomedemstat (MK3543) in Combination with Ruxolitinib in Patients with Myelofibrosis von Gill, Harinder, Au, Lester, Leung, Garret Man Kit, Tsai, Dorothy, Yim, Rita, Chin, Lynn, Li, Vivian, Lee, Paul, Leung, Rock Yuk Yan, Lee, Elaine, Rienhoff Jr, Hugh Young, Kwong, Yok Lam

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Antimalarial iron chelator, FBS0701, shows asexual and gametocyte Plasmodium falciparum activity and single oral dose cure in a murine malaria model von Ferrer, Patricia, Tripathi, Abhai K, Clark, Martha A, Hand, Carla Cerami, Rienhoff, Jr, Hugh Young, Sullivan, Jr, David J

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Bomedemstat (IMG-7289), an LSD1 Inhibitor, Manages the Signs and Symptoms of Essential Thrombocythemia (ET) While Reducing the Burden of Cells Homozygous for Driver Mutations von Goethert, Joachim R., Gill, Harinder, Palandri, Francesca, Ross, David M., Cochrane, Tara, Larsen, Stephen R., Halpern, Anna B., Shortt, Jake, Rossetti, James M., Liang, James, Marchetti, Monia, Wilson, Andrew J., Innes, Andrew, Hanna, Merit, Stevenson, William S., Vannucchi, Alessandro M., Kleppe, Maria, Natsoulis, Georges, Harrison, Claire N, Rienhoff Jr, Hugh Young

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A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload von Neufeld, Ellis J., Galanello, Renzo, Viprakasit, Vip, Aydinok, Yesim, Piga, Antonio, Harmatz, Paul, Forni, Gian Luca, Shah, Farrukh T., Grace, Rachael F., Porter, John B., Wood, John C., Peppe, Jennifer, Jones, Amber, Rienhoff, Hugh Young

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Reflections on my daughter's DNA von Rienhoff, Hugh Young

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Two monogenic disorders masquerading as one: severe congenital neutropenia with monocytosis and non-syndromic sensorineural hearing loss von Venugopal, Parvathy, Gagliardi, Lucia, Forsyth, Cecily, Feng, Jinghua, Phillips, Kerry, Babic, Milena, Poplawski, Nicola K, Rienhoff, Jr, Hugh Young, Schreiber, Andreas W, Hahn, Christopher N, Brown, Anna L, Scott, Hamish S

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Genomewide Association Studies and Assessment of Risk of Disease von Pandey, Janardan P, Rienhoff, Hugh Young, Manolio, Teri A

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Iron-overload-related disease in HFE hereditary hemochromatosis von Rienhoff, Jr, Hugh Young

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Fast approximation to pixelwise relaxivity maps: Validation in iron overloaded subjects von Meloni, Antonella, Zmyewski, Heather, Rienhoff, Hugh Young, Jones, Amber, Pepe, Alessia, Lombardi, Massimo, Wood, John C

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Iron-Overload–Related Disease in HFE Hereditary Hemochromatosis von Waalen, Jill, Beutler, Ernest

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A KDM1A INHIBITOR AND ITS USE IN THERAPY von CELATKA, Cassandra, CLARE, Michael, TAPPER, Amy E, RIENHOFF, Hugh Young Jr, MCCALL, John M

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KDM1A INHIBITORS FOR THE TREATMENT OF DISEASE von CLARE, Michael, RIENHOFF, Jr. Hugh Young, MCCALL, John M

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