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.

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.

The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died, but that won't cause the study to be halted. The FDA has allowed the ...

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.