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European Commission grants marketing authorization for NEZGLYAL® (leriglitazone), the first pharmacological treatment approved for cerebral adrenoleukodystrophy (cALD), a rare neurodegenerative disease

Neuraxpharm Group and Minoryx Therapeutics, today announce that the European Commission (EC) has granted marketing authorization under exceptional circumstances for NEZGLYAL® (leriglitazone), an orally bioavailable, brain penetrating, selective PPAR gamma agonist, as a treatment for male cALD patients, aged 2-12 years with Gadolinium (Gd)-negative brain lesions.

26 Sep 2026

AI correction tone influences effectiveness in combating medical misinformation

At a time of rampant medical misinformation online, artificial intelligence can do a good job of correcting falsehoods, but the tone of the correction is key, according to new research led by Washington State University.

25 Sep 2026

UC researcher receives funding to study bone complications in people with sickle cell disease

September is National Sickle Cell Awareness Month, and a University of Cincinnati College of Medicine hematology researcher has received new grant funding to study one of the disease's painful, under-recognized complications: bone damage.

25 Sep 2026

Study explains how stroke promotes aggressive glioma tumor growth

Researchers at Texas Children's Duncan Neurological Research Institute (Duncan NRI), Baylor College of Medicine and collaborating institutions show in Nature Cancer that stroke promotes glioma growth and uncovered cellular and molecular mediators involved in the process, linking brain injury to malignant brain tumors.

25 Sep 2026

Blood proteins may help monitor Alzheimer’s treatment response

Patients with early symptoms of Alzheimer's disease may be prescribed anti-amyloid therapy, which can help clear the sticky clumps of amyloid protein in the brain that characterize Alzheimer's.

25 Sep 2026

Uterus transplantation shows improving outcomes and successful births nationwide

A decade of uterus transplantation in the United States has resulted in dozens of healthy births and steadily improving surgical outcomes, according to a new national review led by researchers from the Perelman School of Medicine at the University of Pennsylvania.

24 Sep 2026

GLP-1s linked to nutritional deficiencies in children

Children prescribed GLP-1 medications for weight loss, prediabetes, or type 2 diabetes are at risk for nutritional deficiencies – diagnosed in nearly one in six patients (16.8%) within the first year – according to a new study published in the journal Childhood Obesity.

24 Sep 2026

Early treatment may improve outcomes in Shwachman-Diamond syndrome

More than 90% of children born with the rare bone marrow condition called Shwachman-Diamond Syndrome (SDS) survive beyond age 20. But fewer than 30% live beyond age 50.

23 Sep 2026

A child's gut microbiome may offer early clues to type 1 diabetes risk

Researchers tracking 887 children at high genetic risk of type 1 diabetes identified three distinct patterns of gut microbiome maturation during early childhood. One pattern was associated with substantially higher risk of islet autoantibody seroconversion or type 1 diabetes, while host genetic background altered the risk associated with another pattern.

23 Sep 2026

Discovery of receptor-free signaling reveals better targets for treating autoimmune diseases

Our understanding of how cells interact with each other has been driven for decades by the central concept of receptor-mediated signal transduction.

23 Sep 2026

Overheard adult speech shapes word learning in young children

Overhearable speech (speech that may occur when caregivers are talking to each other in the child's presence), constitutes over half of children's language exposure, but its role in language development remains underexplored.

23 Sep 2026

Dietary changes can prevent Hirschsprung disease symptoms

A multi-institutional study led by researchers at Children's Hospital of Philadelphia (CHOP) discovered for the first time that dietary interventions might prevent inflammatory symptoms associated with Hirschsprung disease, a bowel motility disorder.

23 Sep 2026

Early childhood BMI trajectories predict later cardiometabolic health risks

Childhood obesity predicts later cardiometabolic disease, but single-time weight measures may miss dynamic growth information.

23 Sep 2026

Study finds no causal link between prenatal paracetamol and neurodevelopmental disorders

Various studies over the years have linked prenatal use of the pain reliever paracetamol (acetaminophen) with neurodevelopmental disorders such as autism and attention-deficit/hyperactivity disorder (ADHD) in children, but many of the studies were small, poorly designed, or found only weak associations

23 Sep 2026

NCI awards $3.46 million grant to study resistance to acute myeloid leukemia treatment

A five-year, $3.46 million grant from the National Cancer Institute recently was awarded to The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) and the University of California San Francisco (UCSF).

23 Sep 2026

New slow-release drug delivery system provides weeks of pain-numbing anesthetic effects

Most local anesthetics used in medicine only last eight to 12 hours or a day tops. Now Boston Children's Hospital researchers have formulated a more effective slow-release injectable drug delivery system that achieved pain-numbing anesthetic effects lasting two to three weeks in rats compared to a commercial formulation lasting about four to eight hours.

23 Sep 2026

Could a parent’s hospital job shape a child’s nasal microbiome?

A pilot study of 37 families found that children with healthcare-worker parents had lower nasal microbial diversity, with the largest differences among children whose two parents worked in healthcare. Porphyromonas was significantly less abundant, while Neisseria showed a similar non-significant pattern, but the cross-sectional study cannot establish causality or determine whether these microbiota differences affect health.

23 Sep 2026

Children of single mothers by choice show favorable birth outcomes

The children of single mothers who conceive through medically assisted reproduction (MAR) have similar outcomes to babies of partnered mothers who had conceived naturally, a UCL-led study has found.

22 Sep 2026

Ketamine-related bladder problems reported among young teenagers

Non-prescribed ketamine use may start at the age of 13, suggest data from a UK clinic dedicated to the treatment of bladder problems associated with misuse of the drug and published in a clinical letter in the Archives of Disease in Childhood.

22 Sep 2026

A hidden measles signal may reveal how large the next outbreak could be

Researchers used historical measles surveillance and demographic data from pre-vaccination England and Wales to reconstruct how the susceptible population changed between epidemics. Their model linked susceptibility at the start of an epidemic year with the magnitude of the following outbreak, pointing to a potential route for one-year-ahead forecasting that requires further development for modern surveillance settings.

22 Sep 2026