Results 201 to 210 of about 288,749 (359)

Neonatal screening for spinal muscular atrophy: A pilot study in Brazil. [PDF]

open access: yesGenet Mol Biol, 2023
Oliveira Netto AB   +18 more
europepmc   +1 more source

Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double‐Blinded, Placebo‐Controlled, Phase 3 Trial

open access: yesAmerican Journal of Hematology, EarlyView.
ABSTRACT Primary autoimmune thrombocytopenia (ITP) is characterized by thrombocytopenia, bleeding, and reduced health‐related quality of life. In the Phase 3 ADVANCE IV study, intravenous efgartigimod induced significant platelet count responses versus placebo in patients with chronic ITP. ADVANCE SC, a Phase 3, multicenter, randomized, double‐blinded,
Nichola Cooper   +546 more
wiley   +1 more source

Rapid Genome and Exome Sequencing in Inpatients: Clinical Impact at a Tertiary Academic Medical Center

open access: yesAmerican Journal of Medical Genetics Part A, EarlyView.
ABSTRACT The objective of this study is to describe outcomes of rapid exome (rES) and rapid genome sequencing (rGS) in an inpatient setting. This is a retrospective cohort of inpatients with rES or rGS during their hospitalization between April 2016 and November 2023.
Cecilia M. Kessler   +5 more
wiley   +1 more source

Neonatal screening for primary and central congenital hypothyroidism: is it time to go Dutch? [PDF]

open access: yesEur Thyroid J, 2023
Boelen A   +3 more
europepmc   +1 more source

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