Marijuana extract shown to cut life-threatening seizures in half for children with severe epilepsy. A landmark clinical trial has revealed that a drug derived from medical marijuana can significantly ...

The drug was finally approved in September 2025 through the FDA’s accelerated approval pathway, based on improvement in knee extensor muscle strength, for adults and children with Barth syndrome who ...

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

The day we discovered that our then 15-month-old baby was one of just 200 people in the world with a genetic brain condition will be for ever etched on my mind.

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the Committee for Medicinal Products for Human Use (CHMP) of ...

Twelve-year-old Canberra girl Ashley Clifford and her family thought she was one of only two children in Australia with the very rare Beck-Fahrner syndrome. Your essential national news digest: all ...

This Ice Age pair of hunter-gatherers were both shorter than average for their population – one of them markedly so – and now we finally know why. A team of researchers from Europe re-examined the ...

How innovative technologies like AI and gene editing are advancing rare disease drug discovery and development.

Researchers show two distinct MECP2 mutations in Rett syndrome elevate miRNA-126-3p, lowering ZO-1 and weakening blood-brain ...

Evox Therapeutics Ltd ("Evox" or the "Company"), a biotechnology company developing innovative therapies for genetically driven neurodegenerative diseases using a next-generation gene editing modality ...

MIT researchers have discovered that two common genetic mutations that cause Rett syndrome each set off a molecular chain of events that compromises the structural integrity of developing brain blood ...