#rare-disease

Researchers used tiny retinas grown in the lab to show how subtle changes in a growth-controlling protein may drive a rare condition that causes serious eye defects from birth.

Neurocrine Biosciences is buying Soleno Therapeutics for $2.9 billion, gaining an approved treatment for a rare disease associated with insatiable hunger.

An opinion essay on mitochondrial disease argues that medical nutrition is essential to keeping some patients healthy, yet insurers still often treat it as optional rather than medically necessary.

Tanabe Pharma says it has posted positive Phase 3 data for an oral drug targeting two rare disorders associated with pain on light exposure, marking a major clinical milestone after its Bain Capital acquisition.

A correspondence in Nature Medicine argues that bespoke pediatric gene therapies are arriving faster than the traditional drug model can handle, and calls for a new regulatory framework built for individualized medicines

Prime Medicine reverses course to seek FDA approval for a previously sidelined rare immune disease therapy, citing the agency's evolving stance on rare diseases.