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Results from an international clinical trial found that risdiplam, a drug used to treat the rare neurodegenerative disorder Spinal Muscular Atrophy, is safe and effective in newborns.

A multi-institutional team of researchers has trained AI to understand the “language” of proteins and design peptide drugs to target disease proteins – a game-changer for hard-to-treat diseases.

An NIH-funded trial found that two IV doses of dalbavancin, given a week apart, were as safe and effective as 4–6 weeks of standard IV antibiotics for complicated Staphylococcus aureus bloodstream infections.

In the phase 2 SunRISe-1 trial, TAR-200, a slow-release intravesical device delivering gemcitabine, eliminated tumors in 82% of high-risk NMIBC patients unresponsive to BCG therapy. Nearly half remained cancer-free after 12 months.

Researchers have identified a genetic biomarker that predicts whether multiple sclerosis (MS) patients will respond particularly well to treatment with glatiramer acetate. The results come from a multicentre analysis of more than 3,000 MS patients.

Researchers have developed a new engineered protein-based therapy, which acts like a sponge to soak up carbon monoxide from the blood. The molecule appears promising as an antidote for carbon monoxide poisoning with fewer side effects.

A study found that benztropine, a drug used to manage tremors in patients with Parkinson’s, can dramatically reduce levels of tuberculosis-causing bacteria by boosting the body’s natural immune response.

A new global study reveals that male researchers have higher paper retraction rates than their female peers. We spoke to the lead author to find out why this could be the case.

Researchers at the University of California Davis Health have identified a natural compound made by gut bacteria that reversed liver damage and repaired the gut lining in mice after exposure to aflatoxin, a toxin from mold found in foods.

A cancer drug could enhance how patients respond to chemotherapy even in treatment-resistant tumours. In preclinical models, it has already shown promise in making chemotherapy-resistant cancers more responsive to therapy.