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An AI-enabled drug discovery approach identified a potentially game-changing treatment for Rett syndrome, which has been advanced from the lab bench to an FDA Orphan Drug Designation in record time.

Researchers have successfully grown miniature lungs from stem cells – complete with their own functioning blood vessel networks.

In the early 2000s a clinical trial showed that lifestyle modification was better than metformin at preventing Type 2 diabetes. In a follow-up study, health benefits from lifestyle intervention persisted for more than 20 years later.

Putting the brakes on an enzyme might rescue neurons that are dying in Parkinson's disease, stabilizing the progression of symptoms.

Researchers identified a protein that could be targeted to block chronic inflammation. Crucially, this strategy would not interfere with acute inflammation, allowing the immune system to continue responding appropriately to short-term threats.

Researchers tested a gene therapy that restored partial hearing in patients with OTOF gene mutations causing congenital deafness. The therapy delivered a functional OTOF gene using an adeno-associated virus (AAV) injection.

Researchers have identified two proteins in an organelle specific to Plasmodium falciparum that could be used as drug targets for novel antimalarials.

Researchers have pioneered a novel 3D platform that mimics the human bone marrow and immune environment, enabling more predictive testing of cancer immunotherapy success in patients, including CAR T cell therapies.

An oral drug called MA-5 can improve both heart and muscle problems in Barth syndrome. Testing the drug on cells from Barth syndrome patients and in fruit fly models of the disease, the drug boosted cellular energy production by up to 50%.

Targeting the flagellum to deliver a critical, but not fatal, blow to bacteria could help in the fight against antibiotic resistance.