DAYBUE, a synthetic analog of insulin-like growth factor 1, is designed to treat Rett syndrome, although its exact therapeutic mechanism is not fully understood. It is available as an oral ...

We remain encouraged by our productive, ongoing discussions with the FDA through the Regenerative Medicines Advanced Therapy or RMAT mechanism ... of patients with Rett syndrome using a minimally ...

At least $65 million slated for 14 of 35 Alzheimer's disease research centers is in limbo, with funding expected to run out ...

such as Rett syndrome or Angelman syndrome, or even more complex neurodevelopmental conditions. It also provides instant medical recommendations, directing patients to specialized departments and ...

It also has a candidate called ACP-2591 with potential in Rett syndrome and Fragile X syndrome ... could be the first drug with an entirely new mechanism of action to reach the market in decades.

Microglia, Pediatric Neurology, Autism Spectrum Disorder, Epilepsy, Neuroinflammation, Neurodevelopment Kaur, H. (2025) The Role of Microglia in Pediatric Neurological Disorders. Neuroscience and ...

In 2024, the field inched further forward, with Wave Life Sciences announcing the first-ever results from the mechanism in humans ... for editing,” such as in cystic fibrosis and Rett syndrome, he ...

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 ...

Phase 2a TREE study assesses safety and efficacy of leriglitazone Minoryx Therapeutics has dosed the first patient in its phase 2a TREE study, which evaluates the safety and efficacy of leriglitazone ...

Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med -- Study supported FDA and Health Canada ...

A research team led by Mount Sinai has uncovered mechanisms of abnormal immune cell function that may lead to Crohn's disease ...