JanetLKwiatkowskiMD

PubMed

• 1 citations
  Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open...
  Janet L Kwiatkowski, Mark C Walters, Suradej Hongeng, Evangelia Yannaki, Andreas E Kulozik
  Lancet. 2024-11-30
• 1 citations
  Real-world evidence: Long-term safety of deferiprone in a large cohort of patients with sickle cell disease enrolled in a registry for up to 10 years.
  Janet L Kwiatkowski, Alexis A Thompson, Fernando Tricta, Noemi Toiber Temin, Anna Rozova
  American Journal of Hematology. 2024-06-01
• Diagnosis and management of pyruvate kinase deficiency: international expert guidelines.
  Hanny Al-Samkari, Nadine Shehata, Kelly Lang-Robertson, Paola Bianchi, Andreas Glenthøj
  The Lancet. Haematology. 2024-03-01

Journal Articles

• The Phenotypic Spectrum of Germline YARS2 Variants: From Isolated Sideroblastic Anemia to Mitochondrial Myopathy, Lactic Acidosis and Sideroblastic Anemia 2  
  Matthew M Heeney, Gregory A Hale, David P Steensma, Mark D Fleming, Colin A Sieff, Lee M Hilliard, Sylvia S Bottomley, Janet L Kwiatkowski, Haematologica

Abstracts/Posters

• Pyruvate Kinase (PK) Protein and Enzyme Levels in the Diagnosis and Clinical Phenotype of PK Deficiency
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
• Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent B...
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
• Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent _-Thalassemia and Either a _0 or IVS-I-110...
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
• Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/7/2019
• Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/7/2019
• Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/8/2019
• Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/7/2019
• Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent _-Thalassemia in the Northstar (HGB-204) Study
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
• Characterization of the Severe Phenotype of Pyruvate Kinase Deficiency
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/7/2019
• Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent _-Thalassemia in the Northstar (HGB-204) Study
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
• Northstar-2: Updated Safety and Efficacy Analysis of Lentiglobin Gene Therapy in Patients with Transfusion-Dependent _-Thalassemia and Non-_0/_0 Genotypes
  Janet L. Kwiatkowski, 61st Annual American Society of Hematology Meeting, Orlando, FL, 12/9/2019
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Lectures

• ¤-Thalassemia: Expert Insights on New Therapies for an Old Disease 
  61st Annual American Society of Hematology Meeting - Orlando, FL - 12/7/2019
• Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent _-Thalassemia in the Northstar (HGB-204) Study 
  61st Annual American Society of Hematology Meeting - Orlando, FL - 12/7/2019
• Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial 
  2018 ASH Annual Meeting - San Diego, CA - 12/1/2018
• Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar Hgb-204 Study 
  2018 ASH Annual Meeting - San Diego, CA - 12/1/2018
• Thalassemia Management Checklists: Quick Reference Guides to Reduce Disparities in the Care of Patients with Transfusion-Dependent Thalassemia 
  2018 ASH Annual Meeting - San Diego, CA - 12/1/2018
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