| 1 |
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency
Donald B. Kohn et al. · New England Journal of Medicine
Open access |
11 May 2021 |
225 |
| 2 |
Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease
James F. Casella et al. · JAMA
Open access |
20 Apr 2021 |
42 |
| 3 |
Recombinant von Willebrand factor and tranexamic acid for heavy menstrual bleeding in patients with mild and moderate von Willebrand disease in the USA (VWDMin): a phase 3, open-label, randomised, crossover trial
Margaret V. Ragni et al. · The Lancet Haematology
Open access |
26 Jun 2023 |
40 |
| 4 |
L‐leucine improves anemia and growth in patients with transfusion‐dependent Diamond‐Blackfan anemia: Results from a multicenter pilot phase I/II study from the Diamond‐Blackfan Anemia Registry
Adrianna Vlachos, Evangelia Atsidaftos, Mohammad Lutfi Lababidi et al. · Pediatric Blood & Cancer
Open access |
6 Oct 2020 |
35 |
| 5 |
A Phase II Study of Recombinant Interleukin-2 With or Without Recombinant Interferon-?? in Non-Hodgkin??s Lymphoma. A Study of the Cancer and Leukemia Group B
David B. Duggan, María Teresa Santarelli, Kenneth W. Zamkoff et al. · Journal of Immunotherapy
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Aug 1992 |
23 |
| 6 |
Switch to low-fat diet improves outcome of acute lymphoblastic leukemia in obese mice
Jonathan Tucci, Waseem Alhushki, Ting Chen et al. · Cancer & Metabolism
Open access |
16 Oct 2018 |
21 |
| 7 |
Fibrinogen concentrate for bleeding in patients with congenital fibrinogen deficiency: Observational study of efficacy and safety for prophylaxis and treatment
Joseph L. Lasky, Jerome M. Teitel, Michael Wang et al. · Research and Practice in Thrombosis and Haemostasis
Open access |
11 Oct 2020 |
17 |
| 8 |
Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent/young adult patients
Naveen Pemmaraju, Branko Cuglievan, Joseph A. Lasky et al. · eJHaem
Open access |
Feb 2024 |
13 |
| 9 |
Pediatric Patients With Sickle Cell Disease at a Public Hospital: Nutrition, Compliance and Early Experience With L-Glutamine Therapy
Moran Gotesman, Guy Elgar, Laura Hernandez Santiago et al. · In Vivo
Open access |
2022 |
9 |
| 10 |
Improving Preventive Care for Children With Sickle Cell Anemia: A Quality Improvement Initiative
Michael D. Cabana, Anne Marsh, Marsha J. Treadwell et al. · Pediatric Quality and Safety
Open access |
28 Dec 2020 |
6 |
| 11 |
Approach to the Treatment of Expansile Metacarpal Aneurysmal Bone Cysts: A Report of Two Cases
Christopher R. Gajewski, Waseem Alhushki, Prosper Benhaim et al. · Journal of Hand Surgery Global Online
Open access |
4 Jun 2023 |
3 |
| 12 |
P521: TREATMENT OF BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM IN PEDIATRIC PATIENTS WITH TAGRAXOFUSP, A CD123-TARGETED THERAPY
Naveen Pemmaraju, Branko Cuglievan, Joseph L. Lasky et al. · HemaSphere
Open access |
Jun 2022 |
2 |
| 13 |
Concurrent Presentation of von Willebrand Disease and Inflammatory Bowel Disease—A Case Report and Literature Review
Suraj Pratap, Aimee Foord · eJHaem
Open access |
13 Mar 2026 |
1 |
| 14 |
Pancreaticoduodenectomy in 11-year-old male with a non-functional pancreatic neuroendocrine tumor
Whitney Elks, Jessa Rose Li, Alexander Zajack et al. · Journal of Pediatric Surgery Case Reports
Open access |
12 Nov 2022 |
1 |
| 15 |
Robotic enucleation of a solid pseudopapillary tumor in a pediatric patient
Mary Froehlich, Penelope Herder, Alan K. Ikeda et al. · Journal of Pediatric Surgery Case Reports
Open access |
16 May 2022 |
1 |
| 16 |
ID #337 Peripheral Primitive Neuroectodermal Tumor in a Pediatric Patient
Sumit Gupta, Jessica Casey, Joseph Lasky · Neuro-Oncology Pediatrics
Open access |
23 Jun 2026 |
0 |
| 17 |
Immune Effector Cell and Gene Therapies in Pediatric Critical Care
Dristhi S. Ragoonanan, Kris M. Mahadeo
Book chapter |
2026 |
0 |
| 18 |
205P Nirogacestat pediatric compassionate use program: Cross-sectional data review
Barbara J. Brennan, Kelly Vallance, A.B. Oton et al. · ESMO rare cancers.
Open access |
Mar 2026 |
0 |
| 19 |
Immunoliposome for Ewing Sarcoma
DANIEL E. PANOSYAN, WILLIAM S. PANOSYAN, DARON S. YACOUBIAN et al. · Cancer Genomics & Proteomics
Open access |
28 Oct 2025 |
0 |
| 20 |
Allogeneic Hematopoietic Stem Cell Transplant for an Unknown Inherited Bone Marrow Failure Syndrome With a Novel Compound Heterozygous Mutation
Olayinka Okeleji, Sajad Jawad Khazal, Kris Michael Mahadeo et al. · Pediatric Blood & Cancer
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19 May 2025 |
0 |
| 21 |
Neuromuscular Patients Are 40% More Likely to Get a Cast Injury
David G. Stewart · Journal of Pediatric Orthopaedics
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16 Jun 2023 |
0 |
| 22 |
Multicenter, Randomized Crossover Trial Comparing Recombinant Von Willebrand Factor and Tranexamic Acid for Heavy Menstrual Bleeding in Von Willebrand Disease
Margaret V. Ragni et al. · Blood
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15 Nov 2022 |
0 |
| 23 |
Poster: AML-392 Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm in Pediatric Patients With Tagraxofusp, a CD123-Targeted Therapy
Naveen Pemmaraju, Branko Cuglievan, Joseph A. Lasky et al. · Clinical Lymphoma Myeloma & Leukemia
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20 Sep 2022 |
0 |
| 24 |
Inhibit Clinical Trials Platform to Prevent and Eradicate Inhibitors: Feasibility Survey of Current Prophylaxis and Immune Tolerance Practices
Margaret V. Ragni et al. · Blood
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4 Nov 2020 |
0 |
| 25 |
Long-term treatment with elosulfase alfa has an acceptable safety profile for patients with Morquio syndrome type A: Real-world results from the Morquio A Registry Study (MARS)
Barbara K. Burton, Nathalie Guffon, Uma Ramaswami et al. · Molecular Genetics and Metabolism
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30 Jan 2020 |
0 |
| 26 |
Long-term clinical outcomes of patients treated with elosulfase alfa: Five-year real-world results from the Morquio A Registry Study (MARS)
John J. Mitchell, Uma Ramaswami, Nicola Longo et al. · Molecular Genetics and Metabolism
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30 Jan 2020 |
0 |