Results 181 to 190 of about 448,338 (267)

Correction of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion‐Dependent β‐Thalassemia

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT Exagamglogene autotemcel (exa‐cel) is a one‐time, ex vivo, CRISPR‐Cas9 gene edited cell therapy approved for patients with transfusion dependent β‐thalassemia (TDT) aged 12–35 years. In a Phase 3 study (CLIMB THAL‐111), exa‐cel treatment resulted in reactivation of fetal hemoglobin and increases in total hemoglobin, leading to transfusion ...
Sujit Sheth   +25 more
wiley   +1 more source

Interventions for preventing unintended pregnancies among adolescents. [PDF]

open access: yesCochrane Database Syst Rev
Oringanje C   +5 more
europepmc   +1 more source

Guest Editorial: Frontiers of Separation Analysis

open access: yesAnalytical Sciences
Toshio, Takayanagi   +3 more
openaire   +2 more sources

Pediatric Anesthesia: Excellence in Patient Care. [PDF]

open access: yesJ Clin Med
Marhofer P, Zadrazil M, Opfermann P.
europepmc   +1 more source

Lymphatic Abnormalities in Noonan Syndrome Extend Beyond Clinically Apparent Disease

open access: yesAmerican Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Lymphatic disease represents a well‐described manifestation of Noonan syndrome (NS), yet the full phenotypic spectrum remains incompletely characterized, especially in asymptomatic individuals. We conducted a cross‐sectional study including 10 individuals with NS (four with peripheral lymphedema and six without) and 10 age‐ and sex‐matched ...
Inger Norlyk Sheyanth   +7 more
wiley   +1 more source

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