Results 51 to 60 of about 1,206,149 (284)

Idiopathic Infantile arterial calcification –A Very rare case [PDF]

, 2010
A rare case of Idiopathic Arterial Calcification of Infancy (IACI), inherited as an autosomal recessive disease, is ...
Joshi, PS, Sarmila, N, Vani, R
core  

CPX‐351 in High‐Risk Relapsed Pediatric Acute Leukemia: Real‐World Phase 1 Data Establishing the FDA‐Approved Dose

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background/Objectives Outcomes for pediatric relapsed/refractory (R/R) acute myeloid leukemia (AML) remain dismal. CPX‐351, a liposomal formulation of cytarabine and daunorubicin, may have less off‐target toxicities than traditional chemotherapies and has shown improved outcomes for adults with newly diagnosed therapy‐related AML.
Jonathan D. Bender, Lauren Pommert, Lori R. Backus, Andrea Bidwell, Courtney Blank, Erin H. Breese, Karen C. Burns, Adam Lane, Lynn H. Lee, Benjamin Mizukawa, Robin E. Norris, Maureen M. O'Brien, Daniel Peck, Christine Phillips, Thomas D. Ryan, Jennifer Young, Michael Absalon, John P. Perentesis   +17 more
wiley   +1 more source

Retrospective chart review of inherited and idiopathic dystonia

Frontiers in Genetics
Dystonia prevalence and presentation varies both ethnically and geographically. There is a paucity of data on the clinical presentation of dystonia patients in Saudi Arabia and among Arabs.
A. Alakkas, H. Shinawi, J. A. Bajwa, O. Alsinaidi, A. Al-Hashim   +4 more
doaj   +1 more source

A nonhuman primate model of inherited retinal disease. [PDF]

, 2019
Inherited retinal degenerations are a common cause of untreatable blindness worldwide, with retinitis pigmentosa and cone dystrophy affecting approximately 1 in 3500 and 1 in 10,000 individuals, respectively.
Artemyev, Nikolai O, Boyd, Kimberly, Cameron, Ashley, Chen, Rui, Davis, Sarah, Garzel, Laura, Gopalakrishna, Kota N, Harris, R Alan, Huang, Yijun, Kim, Soohyun, Li, Yumei, Liang, Qingnan, Moshiri, Ala, Murphy, Christopher J, Pomerantz, Ori, Raveendran, Muthuswamy, Roberts, Jeffrey, Rogers, Jeffrey, Stout, J Timothy, Thomasy, Sara M, Wang, Jun, Yiu, Glenn   +21 more
core  

Incidence and Risk Factors of Serious Osteonecrosis in Pediatric Acute Lymphoblastic Leukemia: A CYP‐C Population‐Based Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Osteonecrosis (ON) is a debilitating complication of acute lymphoblastic leukemia (ALL) therapy. While numerous studies have explored its incidence and associated risk factors, investigations using large‐scale cohorts remain important to characterize ON across heterogeneous populations.
Noémie de Villiers, Chantal Éthier, Caroline Laverdière, Nathalie Alos, Thai Hoa Tran, Marie‐Claude Pelland‐Marcotte   +5 more
wiley   +1 more source

Lipid abnormalities in foam cell reticulosis of mice, an analogue of human sphingomyelin lipidosis

Journal of Lipid Research, 1969
The lipid changes in the inheritable foam cell reticulosis of mice discovered by Lyons, Hulse, and Rowe have been reexamined. The major abnormality in thymuses from homozygous-abnormal animals has been identified as an increase in the concentration (per ...
Donald P. Fredrickson, Howard R. Sloan, Carl T. Hansen   +2 more
doaj   +1 more source

The natural history of glycogen storage disease type Ib in England: A multisite survey

JIMD Reports, 2021
Glycogen storage disease type Ib (GSDIb) is characterized by hepatomegaly and fasting hypoglycaemia as well as neutropaenia and recurrent infections. We conducted a retrospective observational study on a cohort of patients with GSDIb across England.
Rebecca Halligan, Fiona J. White, Bernd Schwahn, Karolina M. Stepien, Nazreen Kamarus Jaman, Mel McSweeney, Steve Kitchen, Joanna Gribben, Charlotte Dawson, Katherine Lewis, David Cregeen, Helen Mundy, Saikat Santra   +12 more
doaj   +1 more source

Polish business associations: flattened civil society or super lobbies? [PDF]

, 2002
This article tests two hypotheses about post-communist business associations. The first predicts weak business associations which are presented with insurmountable collective action problems by the flattened civil society inherited from totalitarianism ...
Bobbio, Cohen, Draus, Dzwończyk, Gardawski, Gellner, Gerskovits, Hausner, Iain McMenamin, Kolankiewicz, Kozek, Lanzalaco, Linz, Michnik, Moe, Myant, Olson, Olson, Oxhorn, Rostowski, Schmitter, Schmitter, Szawiel, Wyn, Ágh, Łuczkiewicz   +25 more
core   +1 more source

Improving the Evaluation and Management of Transfusion‐Related Iron Overload in Children, Adolescents, and Young Adults Following Cancer Treatment or Hematopoietic Stem Cell Transplantation

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background Transfusion‐related iron overload (TRIO) is a late effect of therapy impacting survivors of childhood cancer and hematopoietic stem cell transplantation (HSCT) who receive frequent packed red blood cell (pRBC) transfusions. Surprisingly, there are no accepted guidelines to assist providers in identifying and treating at‐risk ...
Luke Gingell, Allison Close, Guoli Zhou, Troy C. Quigg   +3 more
wiley   +1 more source

Demographic, clinical, and genetic evaluation of patients with suspected inherited thrombophilia

Van Tıp Dergisi, 2019
INTRODUCTION: Inherited and acquired disorders of hemostasis cause thrombophilia. Thrombosis occurs earlier in individuals with multiple inherited hemostatic disorders.
Pınar Tosun Taşar, Ömer Karaşahin, Özlem Özdemir, Selda Çeneli, Nur Hilal Turgut, Fatih Demirkan   +5 more
doaj   +1 more source