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Journal Articles

T Lymphopenia and Aged Immune System Characterize Immunodeficiency in Adult Patients with Telomere Biology Disorders

Luiz Fernando Bazzo Catto, Nidhi Aggarwal, Ruba Shalhoub, Natthakan Thongon, Tania Rene Machado, Ivana Darden, Jennifer Lotter, Bhavisha A. Patel, Geraldine Aubert, Colin O. Wu, Simona Colla, Cynthia E. Dunbar, Fernanda Rodrigues, Emma M. Groarke

Blood (2024) 144 (Supplement 1): 4094.

https://doi.org/10.1182/blood-2024-207500

Published: 2024

Journal Articles

Danazol Treatment for Telomere Biology Disorders: Long-Term Results of a Phase I/II Study

Nicholas C. Lee, Fernanda Gutierrez-Rodrigues, Ruba Shalhoub, Tania Rene Machado, Jennifer Lotter, Lemlem Alemu, Diego Quinones Raffo, Geraldine Aubert, Colin O. Wu, Bhavisha A. Patel, Neal S. Young, Emma M. Groarke

Blood (2024) 144 (Supplement 1): 439.

https://doi.org/10.1182/blood-2024-194335

Published: 2024

Journal Articles

A Phase I/II, Intra-Patient Dose-Escalation Study of Bitopertin, a Selective Inhibitor of GlyT1 and Heme Synthesis, for Steroid-Refractory Diamond-Blackfan Anemia

David J. Young, Tania Rene Machado, Raymond T. Doty, Colin O. Wu, Janis L. Abkowitz, William Savage, Cynthia E. Dunbar

Blood (2023) 142 (Supplement 1): 1086.

https://doi.org/10.1182/blood-2023-173821

Published: 2023