Neurogene has halted patient enrolment in the high-dose arm of its trial of Rett syndrome gene therapy, sparking another run on its shares. Last week, the company reported that NGN-401 had shown ...

Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share price.

We present the first case-control study addressing both fracture occurrence and fracture mechanisms in Rett syndrome (RTT). Two previous studies have shown increased fracture risk in RTT.

TREE is a phase 2a clinical study assessing the safety and efficacy of lead candidate leriglitazone, a novel, brain-penetrant and selective PPAR gamma agonist, to treat patients with Rett syndrome.

TRUMBULL, Conn., March 13, 2025 (GLOBE NEWSWIRE) -- The Rett Syndrome Research Trust (RSRT), the organization working to cure Rett syndrome, is pleased to announce that Amy Gilliland, President of ...