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Advances in chemistry and delivery are taking oligonucleotide medicines beyond rare diseases

A review in Cell Reports Medicine examines how advances in oligonucleotide chemistry and delivery have established ASOs and RNA interference therapies as a class of genetic medicines, with 21 oligonucleotides approved at the time of writing. It also identifies extrahepatic delivery, safety, target validation, individualized treatment, and data quality as priorities as the field expands into common diseases and explores AI-assisted development.

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

Semaglutide’s heart benefits may extend beyond weight loss

Semaglutide is known to reduce the risk of serious heart disease. However, a new study published in the European Heart Journal today (Thursday) suggests that this effect cannot not be entirely explained by patients losing weight on the drug.

23 Sep 2026

Frailty linked to greater cognitive impairment risk in men with chronic kidney disease

New research from the University of Colorado Anschutz Department of Medicine is shedding light on how women and men with chronic kidney disease (CKD) may fare differently as they age and their disease progresses.

23 Sep 2026

Diabetes medication adherence remains low among disadvantaged patients

A new study led by UCLA Health found that a large percentage of disadvantaged patients are not taking their diabetes medications consistently.

23 Sep 2026

PsychAD studies map molecular mechanisms across major brain disorders

Research led by Mount Sinai scientists within the PsychAD Consortium, a large-scale, multidisciplinary initiative launched in 2019 with support from the National Institute on Aging (NIA), is featured in a landmark collection of nine papers published across the Nature family of journals that maps the molecular mechanisms underlying Alzheimer's disease, Parkinson's disease, schizophrenia, and other brain disorders.

23 Sep 2026

Menopause leaves a blood protein signature linked to brain aging and Alzheimer’s risk

Researchers identified a 16-protein blood signature associated with menopause that tracked more strongly with reproductive hormones than chronological age and was largely replicated in an independent UK Biobank cohort. Higher menopause proteomic scores were also associated with less favorable proteomic aging estimates and poorer cognitive outcomes in older women, including incident Alzheimer’s dementia, although the observational evidence does not establish causality.

23 Sep 2026

Screening approved medicines reveals potential treatments for preterm labor

Shajila Siricilla, PhD, Assistant Professor of Pediatrics, and Jennifer Herington, PhD, Associate Professor of Pediatrics, led a team that systematically screened 1,191 FDA-approved drugs and found that several candidates met criteria supporting further evaluation as potential treatments for preterm labor.

23 Sep 2026

Experimental drug reverses multiple sclerosis damage in mouse model

A drug developed by a UVA Health scientist has not only stopped but reversed paralysis and vision loss in a mouse model of multiple sclerosis.

23 Sep 2026

New AI tool detects signs of endometriosis in milliseconds

Less than a second. That's how long it takes for an emerging AI tool to detect signs of endometriosis through one simple scan.

23 Sep 2026

Cutting ultra-processed foods may improve symptoms of depression

A preliminary study led by UC San Francisco adds to growing evidence that reducing ultra-processed food may improve symptoms of depression.

23 Sep 2026

UVA study evaluates safety and real-world outcomes of histotripsy

UVA Health researchers have reviewed patient outcomes from a new, noninvasive way to treat liver tumors called histotripsy and concluded that while the procedure was associated with low rates of laboratory toxicity and procedural complications, the findings highlight the need to better define which patients are most likely to benefit.

23 Sep 2026

Voluntary exercise reduces stress-linked bladder pain and urinary frequency

Chronic stress may contribute to the pain and urinary symptoms experienced by people with interstitial cystitis/bladder pain syndrome (IC/BPS) by triggering changes in the bladder and nervous system, according to a preclinical study by Weill Cornell Medicine investigators.

23 Sep 2026

Hormonal shifts alter brain patterns linked to female mood

During the menstrual cycle, when progesterone levels are higher, patterns of brain activity emerge that make it more difficult to distinguish between less and more emotionally demanding situations.

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

Genetic system converts scar-forming cells into neurons to restore movement after spinal cord injury

What if cells that form scars after a spinal cord injury could become part of the repair process? Researchers have developed a genetic system that converts certain scar-forming cells into neurons, improving movement in mice and rats with spinal cord injuries.

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

Pharmacist-led reviews improve medication strategies for older cancer patients

There is a pressing need to optimize medication strategies in older adults with cancer, many of whom take ≥5 medications.

23 Sep 2026

Roche advances the Africa Breast Cancer Ambition to power economic growth and transform healthcare across Africa

As global leaders gather for Unstoppable Africa 2026, Roche (www.Roche.com) has reaffirmed its commitment to advancing healthcare as a primary driver of economic resilience across the continent.

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

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Medicine News and Research

Advances in chemistry and delivery are taking oligonucleotide medicines beyond rare diseases

Early treatment may improve outcomes in Shwachman-Diamond syndrome

Semaglutide’s heart benefits may extend beyond weight loss

Frailty linked to greater cognitive impairment risk in men with chronic kidney disease

Diabetes medication adherence remains low among disadvantaged patients

PsychAD studies map molecular mechanisms across major brain disorders

Menopause leaves a blood protein signature linked to brain aging and Alzheimer’s risk

Screening approved medicines reveals potential treatments for preterm labor

Experimental drug reverses multiple sclerosis damage in mouse model

New AI tool detects signs of endometriosis in milliseconds

Cutting ultra-processed foods may improve symptoms of depression

UVA study evaluates safety and real-world outcomes of histotripsy

Voluntary exercise reduces stress-linked bladder pain and urinary frequency

Hormonal shifts alter brain patterns linked to female mood

Early childhood BMI trajectories predict later cardiometabolic health risks

Genetic system converts scar-forming cells into neurons to restore movement after spinal cord injury

Study finds no causal link between prenatal paracetamol and neurodevelopmental disorders

Pharmacist-led reviews improve medication strategies for older cancer patients

Roche advances the Africa Breast Cancer Ambition to power economic growth and transform healthcare across Africa

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

Everything there is to know about biosimilars eBook

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

How Omics Technologies Are Transforming Biological Research and Drug Discovery

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