Study shows that Rett syndrome in females is not just less severe, but different
"That is why it is important to study female mouse models of Rett. They are more relevant for developing treatments." Most ...
pharmaphorum4d
Neurogene shares fall on Rett syndrome gene therapy data
The New York biotech's update focused on data from four patients treated with a low dose of Neurogene's NGN-401 therapy in female Rett syndrome patients, which it said showed "meaningful gains of ...
Nature3mon
Patients With Rett Syndrome Sustain Low-Energy Fractures
We present the first case-control study addressing both fracture occurrence and fracture mechanisms in Rett syndrome (RTT ... A total of 61 female patients with RTT and 122 healthy controls ...
AZoLifeSciences on MSN25d
Minoryx Announces Dosing of the First Patient with Leriglitazone in the Phase 2a Study in Rett Syndrome (TREE Study)
to treat patients with Rett syndrome. The TREE study will enrol 24 female patients aged 5-12 y who will be dosed with ...
Yahoo Finance26d
Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med
The long-term Phase 2/3 study demonstrated DAYBUE had a similar safety profile in children two to four years of age as that observed in individuals with Rett syndrome five years of age and older ...
PharmiWeb27d
Minoryx announces dosing of the first patient with leriglitazone in the phase 2a study in Rett syndrome (TREE study)
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.