Neonatal/pediatrics

Three pediatric brain cancer types share a pineal gland origin

Wed, 11 Mar 2026 12:00:00 -0400

Similarities among three pediatric brain tumors that arise in different structures of the CNS – pineoblastoma, retinoblastoma and Group 3 medulloblastoma – have been linked to their shared origin during pineal gland development. Scientists at St. Jude Children’s Research Hospital have identified the molecular signatures that drive these tumors from pinealocyte progenitor cells that conserve a common differentiation program, providing a shared therapeutic target for these three cancer types.

C-Further unveils pediatric cancer programs

Tue, 10 Mar 2026 09:00:00 -0400

C-Further, an international consortium supporting new therapeutics for pediatric cancers, has unveiled the first early-stage therapeutic programs in its pipeline. The company said it is advancing CF-012 and CF-033 through its collaborative model.

Three pediatric brain cancer types share a pineal gland origin

Tue, 10 Mar 2026 09:00:00 -0400

Targeting TRPM7 ameliorates neonatal HIBI, study finds

Wed, 11 Feb 2026 08:00:00 -0500

Hypoxic-ischemic brain injury (HIBI) is a condition affecting neonates and is a leading global cause of perinatal neurological morbidity, with limited therapeutic options. Regarding its pathogenesis, the ion channel-kinase transient receptor potential melastatin 7 (TRPM7) is a known contributor to HIBI pathology and was the focus of a recently reported study.

β3-Adrenoceptor agonism plays protective role in bronchopulmonary dysplasia

Wed, 28 Jan 2026 08:00:00 -0500

Bronchopulmonary dysplasia (BPD) is a chronic disease of the lungs that mainly affects premature newborns, especially those who receive mechanical ventilation, which can contribute to altering normal lung development. There are few treatment options with limited efficacy to improve the prognosis of BPD.

Succinate prodrug NV-354 protects the brain in complex I deficiency

Tue, 20 Jan 2026 08:00:00 -0500

Leigh syndrome is a fatal pediatric neurodegenerative disorder caused by mitochondrial dysfunction, most often due to defects in the mitochondrial respiratory chain. The Ndufs4 knockout (Ndufs4 KO) mouse is an established model of the disease, as loss of the NDUFS4 subunit leads to complex I (CI) deficiency and reproduces the neurological decline and pathology seen in affected children. Researchers from The Children’s Hospital of Philadelphia Research Institute and collaborators described how NV-354, a water-soluble prodrug of succinate, may mitigate this mitochondrial dysfunction.

Genedx delivers WGS prenatal testing

Wed, 07 Jan 2026 11:00:00 -0500

Genedx Holding Corp. launched Genomedx Prenatal, its whole genome sequencing test, to provide more definitive diagnoses of the causes of fetal abnormalities identified by ultrasound. By combining the company’s decade of experience in prenatal exome testing and its massive Genedx Infinity rare disease dataset, the test can determine not just whether a pregnancy has a risk of a genetic disorder but whether the fetus actually has a genetic disorder.

2025 marks a breakthrough year for in vivo gene therapies

Tue, 30 Dec 2025 08:00:00 -0500

Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them.

2025 marks a breakthrough year for in vivo gene therapies

Tue, 30 Dec 2025 08:00:00 -0500

Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.

2025 marks a breakthrough year for in vivo gene therapies

Mon, 29 Dec 2025 08:00:00 -0500

Crescom wins FDA clearance for bone analysis

Wed, 24 Dec 2025 11:00:00 -0500

Crescom Co. Ltd., an AI musculoskeletal imaging company, gained U.S. FDA 510(k) clearance Dec. 24 for MediAI-BA, its AI-powered pediatric and adolescent bone age analysis software. Classified as a class II medical device, MediAI-BA evaluates bone age and suggests predicted adult height based on growth plate status assessed by hand and wrist X-ray imaging. Prior clinical trial results demonstrated MediAI-BA had specialist-level accuracy, recording a mean absolute deviation (MAD) of 0.39 years.

US Senate ends 2025 with no gift for kids with cancer

Tue, 23 Dec 2025 12:00:00 -0500

After being unanimously passed by the U.S. House Dec. 1, the bipartisan Mikaela Naylon Give Kids a Chance Act seemed to be headed for sure passage in the Senate before it adjourned late last week.

Osteopontin-derived peptide limits lung injury by preserving mitochondria

Tue, 23 Dec 2025 08:00:00 -0500

Bronchopulmonary dysplasia (BPD) remains a significant challenge in neonatal care, particularly affecting preterm infants with low birth weight who often require oxygen therapy or mechanical ventilation to survive.

Medline lines up IPO, Billiontoone hits the road

Wed, 29 Oct 2025 12:00:00 -0400

Medline Inc. filed a public S1 with the SEC Oct. 28, moving the IPO first signaled in December 2024 closer to reality. The Northfield, Ill.-based medical supply distributor did not disclose the number of shares to be offered or their price, but Renaissance Capital expects the IPO to raise about $5 billion, which would make it the largest in any industry this year and one of the largest ever in med tech. Billiontoone Inc. also advanced its IPO, as it began the road show for its public debut in early November.

Illumina's constellation tech a star in Genedx pilot

Wed, 15 Oct 2025 12:00:00 -0400

Genedx LLC partnered with Illumina Inc. to test whether Illumina's constellation map read technology could shed light on hard-to-detect variants that appear in rare diseases. Constellation met or exceeded the ability of other sequencing methods to detect variants implicated in multiple disorders and worked across a range of sample types, a study presented at the American Society for Human Genetics (ASHG) Annual Meeting in Boston on Oct. 15 found.

Pacbio’s Puretarget simplifies carrier screening

Mon, 29 Sep 2025 12:00:00 -0400

Up to 71% of people carry at least one pathogenic variant that could contribute to development of a heritable disorder in offspring, but until now, prospective parents often had to undergo multiple tests to understand their risks. Pacific Biosciences of California Inc. (Pacbio)’s expanded Puretarget portfolio provides a quicker and more streamlined solution as it covers all challenging tier 3 genes identified in the American College of Medical Genetics technical standard.

Storx sensors monitor fetal health during labor and delivery

Fri, 29 Aug 2025 12:00:00 -0400

Researchers from the University of California, Davis (UC-Davis) continue to assemble intellectual property in support of their development of methods and techniques which improve the accuracy of wearable sensor technologies.

AAP unveils its own updated childhood vaccine schedule

Tue, 19 Aug 2025 11:00:00 -0400

For more than 30 years, the U.S. CDC’s Advisory Committee for Immunization Practices (ACIP) and the American Academy of Pediatrics (AAP) worked together to harmonize their evidence-based vaccine schedules. Not anymore.

NrCAM as a predictor of fetal growth restriction

Tue, 29 Jul 2025 09:00:00 -0400

Collaborators in Australia, South Africa, Sweden and the U.K. have linked reduced levels of neuronal cell adhesion molecule (NrCAM) in maternal or placental blood to greater risk of fetal growth restriction in newborns and of preeclampsia in the mothers, based on analysis of various cohorts of patients from several countries.

FDA green lights Cochlear’s smart cochlear implant

Fri, 11 Jul 2025 12:00:00 -0400

Cochlear Ltd. heard good news from the U.S. FDA, as the agency approved its next-generation cochlear implant, the Nucleus Nexa System, the first smart cochlear implant system. Cochlear expects to launch the new products in the U.S. in the next few months, Brendan Murray, vice president for Cochlear Implant products portfolio and strategy told BioWorld.

GC Genome plans $30M IPO to diversify genomic products, suppliers

Tue, 27 May 2025 16:00:00 -0400

GC Genome Corp. priced a Kosdaq offering of 4 million shares at ₩10,500 per share May 27, entailing a gross ₩42 billion (US$30.5 million) raise in early June. The Yongin, South Korea-based genomics subsidiary of GC Biopharma Corp. fixed its shares price to the top of its ₩9,000 to ₩15,000 price band May 27 after conducting demand forecasting on domestic and international institutional investors from May 19 to May 23.

Calla Lily secures £1M for progesterone delivery device

Thu, 03 Apr 2025 12:00:00 -0400

Calla Lily Clinical Care Ltd. secured £1 million (US$1.3 million) in funding from the National Institute for Health and Care Research to begin clinical trials of Callavid, a drug delivery technology which treats women at risk of miscarriage.

AI used to ID abnormalities in unborn babies

Mon, 31 Mar 2025 12:00:00 -0400

The use of an AI tool could speed up the time it takes to do pregnancy scans by almost half and it is still able to identify any abnormalities in the fetus, researchers found. Results from a study showed the AI tool used to assist with 20-week pregnancy scans reduced the scan length by more than 40% and still maintained the same accuracy and reliability of diagnoses.

COVID-19 and Epstein-Barr cause inflammatory shock in children

Thu, 20 Mar 2025 10:00:00 -0400

Multisystem inflammatory syndrome in children (MIS-C), a serious disorder that develops after SARS-CoV-2 infection, could arise from latent infection of another pathogen, the Epstein-Barr virus (EBV). Researchers at Charité – Universitätsmedizin Berlin and the German Rheumatology Research Center (DRFZ) have linked the inflammatory effect of this co-infection with transforming growth factor β (TGF-β), ruling out the possibility that MIS-C is caused by an autoimmune reaction, or persistence of the coronavirus in the body.

Aspargo Companion can spray most meds, alert to missed doses

Wed, 05 Mar 2025 11:00:00 -0500

In an advance that could significantly lighten the load for caretakers in the “sandwich generation” and reduce loneliness in elderly patients, Aspargo Labs Inc. developed a metered delivery device that optimizes absorption of pharmaceuticals and reminds users to take their medications.

Aspargo Companion can spray most meds, alert to missed doses

Tue, 04 Mar 2025 11:00:00 -0500

US FDA clears Sonio Suspect for fetal anomaly detection

Mon, 24 Feb 2025 11:00:00 -0500

The U.S. FDA hasn’t taken up criminal justice as a sideline, but it did just clear Sonio Suspect. Far from a social miscreant, Suspect improves detection of fetal anomalies by 22 points by automatically detecting multiple types of abnormal findings and allows for detection as early as 11 weeks of gestation.

China prioritizes mental health, pediatric care in 2025

Wed, 19 Feb 2025 12:00:00 -0500

China’s National Health Commission is designating a three-year period from 2025 to 2027 as the “years of pediatric and mental health services,” in line with a new pledge to achieve eight initiatives in the health care sector aimed at servicing underserved populations.

US FDA clears Roche’s preeclampsia test

Fri, 14 Feb 2025 11:00:00 -0500

With rates of preeclampsia skyrocketing, the U.S. FDA’s510(k) clearance of Roche Holding AG’s Elecsys test for preeclampsia offers some hope of reducing the number of women and infants who die or experience life-long consequences from the development of dangerously high blood pressure during late pregnancy and in the days immediately following delivery.

Cornerstone Robotics raises $70M to expand international footprint

Tue, 14 Jan 2025 11:00:00 -0500

Cornerstone Robotics Ltd. raised more than $70 million in a series C round to expand its international footprint and accelerate R&D efforts for its robotic surgery solutions. The funds will allow the Hong Kong-headquartered company to accelerate commercialization of new surgical robotic products, conduct clinical trials, obtain regulatory approvals and promote global adoption of advanced robotic surgical solutions.

Neonatal/pediatrics

Three pediatric brain cancer types share a pineal gland origin

C-Further unveils pediatric cancer programs

Three pediatric brain cancer types share a pineal gland origin

Targeting TRPM7 ameliorates neonatal HIBI, study finds

β3-Adrenoceptor agonism plays protective role in bronchopulmonary dysplasia

Succinate prodrug NV-354 protects the brain in complex I deficiency

Genedx delivers WGS prenatal testing

2025 marks a breakthrough year for in vivo gene therapies

2025 marks a breakthrough year for in vivo gene therapies

2025 marks a breakthrough year for in vivo gene therapies

Crescom wins FDA clearance for bone analysis

US Senate ends 2025 with no gift for kids with cancer

Osteopontin-derived peptide limits lung injury by preserving mitochondria

Medline lines up IPO, Billiontoone hits the road

Illumina's constellation tech a star in Genedx pilot

Pacbio’s Puretarget simplifies carrier screening

Storx sensors monitor fetal health during labor and delivery

AAP unveils its own updated childhood vaccine schedule

NrCAM as a predictor of fetal growth restriction

FDA green lights Cochlear’s smart cochlear implant

GC Genome plans $30M IPO to diversify genomic products, suppliers

Calla Lily secures £1M for progesterone delivery device

AI used to ID abnormalities in unborn babies

COVID-19 and Epstein-Barr cause inflammatory shock in children

Aspargo Companion can spray most meds, alert to missed doses

Aspargo Companion can spray most meds, alert to missed doses

US FDA clears Sonio Suspect for fetal anomaly detection

China prioritizes mental health, pediatric care in 2025

​US FDA clears Roche’s preeclampsia test

Cornerstone Robotics raises $70M to expand international footprint

US FDA clears Roche’s preeclampsia test