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BioWorld - Saturday, March 7, 2026
Home » Topics » Disease categories and therapies » Neonatal/pediatrics

Neonatal/pediatrics
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Concept art for prenatal genetic testing and whole genome sequencing.
Genetic/congenital

ASGCT: In utero interventions can prevent organ damage after birth

May 9, 2024
By Mar de Miguel
“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”
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Medically accurate illustration of a baby in the womb of a pregnant mother
Cardiovascular

Peripartum cardiomyopathy is associated with placental senescence

April 24, 2024
By Mar de Miguel
Scientists at Massachusetts General Hospital have linked the risk of heart failure during pregnancy and senescence proteins produced by placental aging, which could clarify how peripartum cardiomyopathy (PPCM) is triggered and opens the door to the development of cardiac function therapies in late pregnancy.
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NICU beanie
Patents

Beanie hat protects hearing of babies in NICUs, encourages development

March 27, 2024
By Simon Kerton
In what represents its first patenting, Sonura LLC has been issued with a patent for an aural device designed to protect the hearing of infants in a neonatal intensive care unit (NICU) by active filtering, while, at the same time, providing aural stimulation for neurological development.
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3D illustration of organoid models in a petri dish
Neonatal/Pediatrics

3D organoids arising from amniotic fluid cells may be a potent prenatal tool

March 8, 2024
By Xavier Bofill Bruna
Organoids are 3D models created from human stem cells and resemble fetal tissues. In an article published in Nature Medicine on March 4, 2024, researchers from University College London provided details on the possibility of generating organoids from epithelial cells collected from amniotic fluid without terminating the pregnancy.
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Neonatal/Pediatrics

3D organoids arising from amniotic fluid cells may be a potent prenatal tool

March 5, 2024
By Xavier Bofill Bruna
Organoids are 3D models created from human stem cells and resemble fetal tissues. In an article published in Nature Medicine on March 4, 2024, researchers from University College London provided details on the possibility of generating organoids from epithelial cells collected from amniotic fluid without terminating the pregnancy.
Read More
Infant examination
Gastrointestinal

Neurenati Therapeutics secures funding to advance candidate for Hirschsprung’s disease

March 1, 2024
Neurenati Therapeutics has closed its seed funding round, securing CA$1.2 million (US$884,000) to advance development of therapies for various rare diseases, including pediatric conditions.
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Brain, illustrating pain/injury
Neurology/Psychiatric

New pediatric rat model of DFP-induced status epilepticus

Feb. 6, 2024
Researchers from Texas A&M University System have detailed the development and characterization of a novel pediatric rat model of organophosphate (OP)-induced status epilepticus (SE).
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Dupixent

US FDA clears Dupixent for children with eosinophilic esophagitis

Jan. 26, 2024
By Karen Carey
Becoming the first treatment for children ages 1 to 11 with eosinophilic esophagitis (EE), Sanofi SA and Regeneron Pharmaceuticals Inc.’s IL-4/IL-13 inhibitor Dupixent (dupilumab) was cleared by the U.S. FDA on Jan. 25.
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Medically accurate illustration of a baby in the womb of a pregnant mother
Obstetrics

Maternal-fetal cellular crosstalk could predict preterm labor

Jan. 11, 2024
By Mar de Miguel
Listening to conversations between a mother and her unborn child on the cellular level could inform how the pregnancy is going and prevent complications. Three scientific groups from the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), in collaboration with Wayne State University and Michigan State University, have used single-cell RNA sequencing techniques to decipher these words, identify the cellular language of these interactions in the placenta and establish a cellular atlas.
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Neurology/Psychiatric

Bloomsbury Genetic Therapies obtains orphan drug designations in US and Europe for BGT-INAD

Oct. 25, 2023
Bloomsbury Genetic Therapies Ltd. has announced it received orphan drug designations from the FDA and the European Commission for BGT-INAD, an investigational gene therapy for the treatment of infantile neuroaxonal dystrophy (INAD).
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