Neurogene expects dosing for a registrational clinical trial of its investigational gene therapy NGN-401 for people with Rett…
Articles by Lindsey Shapiro, PhD
Marvel Biosciences plans to ask the U.S. Food and Drug Administration (FDA) to grant orphan drug status to its…
Treatment with esketamine — a molecule chemically related to the dissociative…
Health Canada has agreed to review Acadia Pharmaceuticals’ application seeking approval of trofinetide — sold in the U.S.
Neurogene is expanding its ongoing Phase 1/2 clinical trial testing gene therapy candidate NGN-401 in girls with Rett…
Two young girls with Rett syndrome have been dosed with Neurogene‘s gene therapy candidate NGN-401 in a Phase…
Long-term treatment with Anavex 2-73 (blarcamesine) provided sustained reductions in disease severity and progression for adult Rett syndrome…
For caregivers of people with Rett syndrome, symptoms related to communication, mobility, and functional hand movements are the most…
Treatment with the diabetes medication metformin for four months led to cognitive improvements and restored brain energy production in…
A so-called positive allosteric modulator aimed at increasing activity at a subset of muscarinic acetylcholine receptors (mAChRs) — called M1…