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Researchers find novel path to treat Rett syndrome

A team of researchers at Texas Children's Duncan Neurological Research Institute (NRI) and Baylor College of Medicine report in Science Translational Medicine a potential new approach to treat Rett syndrome – offering early promise for a rare neurodevelopmental disorder that currently has no cure.

5 Mar 2026

Novel gene regulation therapy passes key clinical milestone

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, show that the first gene regulation treatment for epilepsy is safe and well tolerated by patients with Dravet syndrome for whom antiseizure medications are not effective.

4 Mar 2026

Experimental therapy dramatically reduces seizures in children with Dravet syndrome

A new experimental treatment for children with a hard-to-treat form of epilepsy is safe and can reduce seizures dramatically, helping them lead much healthier and happier lives, the findings of a UCL and Great Ormond Street Hospital-led international clinical trial show.

4 Mar 2026

New fragmentome technology can detect early liver fibrosis and cirrhosis

Researchers at the Johns Hopkins Kimmel Cancer Center report that an artificial intelligence (AI)-based liquid biopsy test using genome-wide cell-free DNA (cfDNA) fragmentation patterns and repeat landscapes can detect early liver fibrosis and cirrhosis, and may also reveal signals of broader chronic disease burden.

4 Mar 2026

Innovative peptide stapling system targets hard-to-treat cancers

Researchers at the University of Bath have developed a new technology that uses bacteria to build, chemically stabilize, and test millions of potential drug molecules inside living cells, making it much quicker and easier to discover new treatments for difficult-to-treat cancers.

3 Mar 2026

New collaboration explores familial inflammatory bowel disease in Saudi Arabia

Mount Sinai and King Saud University Medical City in Riyadh, Saudi Arabia, today announced a three-year collaboration aimed at better understanding why inflammatory bowel disease (IBD) runs in some Saudi families, and how that knowledge can lead to risk ascertainment, earlier diagnosis and more personalized treatment options.

3 Mar 2026

UCF researcher explores insulin signaling as new target for diabetic neuropathy

Neuropathy – chronic pain, numbness and tingling in the hands and feet – is a challenging fact of life for many patients with Type 1 diabetes.

2 Mar 2026

New method isolates true transcription factor targets in tuberculosis bacteria

Inside every cell, thousands of molecular signals collide, overlap, and compensate, obscuring the true drivers of gene expression.

2 Mar 2026

Global breast cancer burden rising fastest in low-income countries

Despite recent advancements in breast cancer treatments, new breast cancer cases in women are predicted to rise by a third globally from 2.3 million in 2023 to more than 3.5 million in 2050.

2 Mar 2026

NIH’s continued investment fuels TMJ pain research

Chronic pain is one of the most common health conditions worldwide. Back pain is the most frequently reported type, followed closely by head and face pain linked to the jaw joint, in the form of temporomandibular joint (TMJ) disorder.

27 Feb 2026

Tick-derived protein discovery can advance treatment for inflammatory diseases

When the immune system detects a harmful or foreign agent it triggers an inflammatory response small proteins called chemokines direct immune cells to the site of the injury or infection, resulting in the invader being inactivated.

27 Feb 2026

Building an affordable toolkit for healthy aging

As people age, they experience a gradual decline in intrinsic capacity (IC), the combined physical and mental abilities that support independence and health. The World Health Organization recognizes IC as a central indicator of resilience in aging, yet no affordable, validated tool exists to measure it.

27 Feb 2026

Extra copies of chromosome 1q may play key role in the earliest stages of pancreatic cancer

Researchers at the Johns Hopkins Kimmel Cancer Center and its Ludwig Center uncovered new evidence that extra copies of a specific chromosome segment - chromosome 1q - may play a key role in the earliest stages of pancreatic cancer development.

26 Feb 2026

Smoking triggers epigenetic damage linked to macular degeneration

Through a series of experiments supported by the National Institutes of Health, Johns Hopkins Medicine (JHM) researchers say they have advanced understanding of how smoking damages the eye and contributes to the development of age-related macular degeneration (AMD), the leading worldwide cause of visual impairment and blindness among people 50 and older.

26 Feb 2026

New method clarifies whether NOTCH1 variants cause congenital heart defects

One to two out of every 100 newborn babies are born with a Congenital Heart Defect (CHD), yet the exact cause remains unclear.

26 Feb 2026

New laser technique ensures integrity of mRNA therapeutics

Messenger RNA (mRNA) technology is transforming medicine by providing our cells with genetic instructions to produce proteins that help the immune system prevent or fight a wide range of diseases, including cancer and other rare disorders.

26 Feb 2026

Novel lipid bubble delivery shows promise for ALS treatment

A researcher at the University of Missouri has made a promising breakthrough in the quest to help people with amyotrophic lateral sclerosis (ALS), the neurodegenerative disorder commonly known as Lou Gehrig's disease.

26 Feb 2026

New daily tablet simplifies long-term HIV care

A new, daily oral tablet that combines two current HIV treatment medications, bictegravir and lenacapavir (BIC/LEN), may be able to effectively replace more complicated HIV treatment regimens used by people living with HIV who are long-term survivors, according to the results of a new phase 3 clinical trial published in The Lancet.

26 Feb 2026

Superagers show greater neuron growth linked to strong memory

Brains of older adults with super healthy cognition grow more new neurons than those of their peers, according to a study from UIC, Northwestern University and the University of Washington.

25 Feb 2026

Early-life immune system differences drive childhood eczema risk

A team of researchers at the Icahn School of Medicine at Mount Sinai, Weill Cornell Medicine, and other institutions have uncovered a key biological explanation for why eczema so often starts in childhood.

25 Feb 2026