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The Inherited Bone Marrow Failure Syndromes

The Pediatric clinics of North America, 2013-12, Vol.60 (6), p.1291-1310

2013

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Chemosensitization to cisplatin by inhibitors of the Fanconi anemia/BRCA pathway

Molecular cancer therapeutics, 2006-04, Vol.5 (4), p.952-961

2006

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Protein degraders enter the clinic — a new approach to cancer therapy

Nature reviews. Clinical oncology, 2023-04, Vol.20 (4), p.265-278

2023

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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

Haematologica (Roma), 2011-04, Vol.96 (4), p.521-525

2011

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Phase 1b study of bavdegalutamide, an androgen receptor PROTACdegrader, combined with abiraterone in patients with metastatic prostate cancer

Journal of clinical oncology, 2022-06, Vol.40 (16_suppl), p.TPS5106-TPS5106

2022

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Abstract PD13-08: First-in-human safety and activity of ARV-471, a novel PROTAC® estrogen receptor degrader, in ER+/HER2- locally advanced or metastatic breast cancer

Cancer research (Chicago, Ill.), 2022-02, Vol.82 (4_Supplement), p.PD13-08-PD13-08

2022

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Practical implications of liver and heart iron load assessment by T2‐MRI in children and adults with transfusion‐dependent anemias

American journal of hematology, 2008-10, Vol.83 (10), p.781-783

2008

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Potent and Orally Bioavailable BCL6 PROTAC TM Degraders Demonstrate Efficacy in Pre-Clinical Models of Diffuse Large B-Cell Lymphoma (DLBCL)

Blood, 2021-11, Vol.138 (Supplement 1), p.2272-2272

2021

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Safety of Gemtuzumab Ozogamicin As Monotherapy or Combination Therapy in an Expanded-Access Protocol for Patients with Relapsed or Refractory Acute Myeloid Leukemia

Blood, 2018-11, Vol.132 (Supplement 1), p.3979-3979

2018

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Deferasirox pharmacokinetics in patients with adequate versus inadequate response

Blood, 2009-11, Vol.114 (19), p.4009-4013

2009

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TERC and TERT gene mutations in patients with bone marrow failure and the significance of telomere length measurements

Blood, 2009-01, Vol.113 (2), p.309-316

2009

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The palatability and tolerability of deferasirox taken with different beverages or foods

Pediatric blood & cancer, 2013-09, Vol.60 (9), p.1507-1512

2013

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Strategies to Improve Iron Chelation In Thalassemia Patients Poorly Responsive to Deferasirox

Blood, 2010-11, Vol.116 (21), p.4279-4279

2010

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The palatability and tolerability of deferasirox taken with different beverages or foods: Palatability and Tolerability of Deferasirox

Pediatric blood & cancer, 2013-09, Vol.60 (9), p.1507-1512

2013

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The Palatability and Tolerability of Deferasirox (Exjade®) Taken with Meals, Different Liquids, or Crushed and Added to Food

Blood, 2010-11, Vol.116 (21), p.5155-5155

2010

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A Phase 1B Dose-Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of FBS0701, a Novel Oral Iron Chelator for the Treatment of Chronic Iron Overload

Blood, 2010-11, Vol.116 (21), p.2057-2057

2010

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Prior Gemtuzumab Ozogamicin Exposure in Adults With Acute Myeloid Leukemia Does Not Increase Hepatic Veno-occlusive Disease Risk After Allogeneic Hematopoietic Cell Transplantation: a CIBMTR Analysis

Biology of blood and marrow transplantation, 2019-12, Vol.26 (5), p.884-892

2019

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Perioperative management of a child with severe hypertension from a catecholamine secreting neuroblastoma

Pediatric anesthesia, 2005-07, Vol.15 (7), p.606-610

2005

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TERC and TERTgene mutations in patients with bone marrow failure and the significance of telomere length measurements

Blood, 2009, Vol.113 (2), p.309-316

2009

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Characterization of Veno-Occlusive Disease (VOD) in Adult Patients (Pts) with Acute Myeloid Leukemia (AML) Receiving Gemtuzumab Ozogamicin (GO) before Allogeneic Stem Cell Transplant (SCT)

Biology of blood and marrow transplantation, 2018-03, Vol.24 (3), p.S301-S302

2018

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434 - Characterization of Veno-Occlusive Disease (VOD) in Adult Patients (Pts) with Acute Myeloid Leukemia (AML) Receiving Gemtuzumab Ozogamicin (GO) before Allogeneic Stem Cell Transplant (SCT)

Biology of blood and marrow transplantation, 2018-03, Vol.24 (3), p.S301-S302

2018

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435 - Veno-Occlusive Disease Characteristics in Pediatric Patients with Acute Myeloid Leukemia Receiving Gemtuzumab Ozogamicin before Allogeneic Stem Cell Transplant

Biology of blood and marrow transplantation, 2018-03, Vol.24 (3), p.S302-S302

2018

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Veno-Occlusive Disease Characteristics in Pediatric Patients with Acute Myeloid Leukemia Receiving Gemtuzumab Ozogamicin before Allogeneic Stem Cell Transplant

Biology of blood and marrow transplantation, 2018-03, Vol.24 (3), p.S302

2018

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Case report: Perioperative management of a child with severe hypertension from a catecholamine secreting neuroblastoma

Pediatric anesthesia, 2005-07, Vol.15 (7), p.606-610

2005

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Practical Implications of Non-Invasive MRI-Based Liver and Cardiac Iron Assessments in Children and Adults with Transfusion-Dependent Anemias

Blood, 2007-11, Vol.110 (11), p.2666-2666

2007

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The Inherited Bone Marrow Failure Syndromes

Chemosensitization to cisplatin by inhibitors of the Fanconi anemia/BRCA pathway

Protein degraders enter the clinic — a new approach to cancer therapy

A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload

Phase 1b study of bavdegalutamide, an androgen receptor PROTACdegrader, combined with abiraterone in patients with metastatic prostate cancer

Abstract PD13-08: First-in-human safety and activity of ARV-471, a novel PROTAC® estrogen receptor degrader, in ER+/HER2- locally advanced or metastatic breast cancer

Practical implications of liver and heart iron load assessment by T2‐MRI in children and adults with transfusion‐dependent anemias

Potent and Orally Bioavailable BCL6 PROTAC TM Degraders Demonstrate Efficacy in Pre-Clinical Models of Diffuse Large B-Cell Lymphoma (DLBCL)

Safety of Gemtuzumab Ozogamicin As Monotherapy or Combination Therapy in an Expanded-Access Protocol for Patients with Relapsed or Refractory Acute Myeloid Leukemia

Deferasirox pharmacokinetics in patients with adequate versus inadequate response

TERC and TERT gene mutations in patients with bone marrow failure and the significance of telomere length measurements

The palatability and tolerability of deferasirox taken with different beverages or foods

Strategies to Improve Iron Chelation In Thalassemia Patients Poorly Responsive to Deferasirox

The palatability and tolerability of deferasirox taken with different beverages or foods: Palatability and Tolerability of Deferasirox

The Palatability and Tolerability of Deferasirox (Exjade®) Taken with Meals, Different Liquids, or Crushed and Added to Food

A Phase 1B Dose-Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of FBS0701, a Novel Oral Iron Chelator for the Treatment of Chronic Iron Overload

Prior Gemtuzumab Ozogamicin Exposure in Adults With Acute Myeloid Leukemia Does Not Increase Hepatic Veno-occlusive Disease Risk After Allogeneic Hematopoietic Cell Transplantation: a CIBMTR Analysis

Perioperative management of a child with severe hypertension from a catecholamine secreting neuroblastoma

TERC and TERTgene mutations in patients with bone marrow failure and the significance of telomere length measurements

Characterization of Veno-Occlusive Disease (VOD) in Adult Patients (Pts) with Acute Myeloid Leukemia (AML) Receiving Gemtuzumab Ozogamicin (GO) before Allogeneic Stem Cell Transplant (SCT)

434 - Characterization of Veno-Occlusive Disease (VOD) in Adult Patients (Pts) with Acute Myeloid Leukemia (AML) Receiving Gemtuzumab Ozogamicin (GO) before Allogeneic Stem Cell Transplant (SCT)

435 - Veno-Occlusive Disease Characteristics in Pediatric Patients with Acute Myeloid Leukemia Receiving Gemtuzumab Ozogamicin before Allogeneic Stem Cell Transplant

Veno-Occlusive Disease Characteristics in Pediatric Patients with Acute Myeloid Leukemia Receiving Gemtuzumab Ozogamicin before Allogeneic Stem Cell Transplant

Case report: Perioperative management of a child with severe hypertension from a catecholamine secreting neuroblastoma

Practical Implications of Non-Invasive MRI-Based Liver and Cardiac Iron Assessments in Children and Adults with Transfusion-Dependent Anemias

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