Browsing Tag
Rett Syndrome
5 posts
Neurogene (NASDAQ: NGNE) advances Rett syndrome gene therapy with Embolden trial launch and positive pediatric data
Neurogene reports progress on NGN-401 Rett syndrome gene therapy with pivotal trial launch and strong pediatric data. See what’s ahead in 2026.
Acadia’s LOTUS study confirms long‑term benefits and manageable side‑effects of DAYBUE over 12 months in Rett syndrome
New real-world data shows DAYBUE improves Rett syndrome symptoms and is tolerable over 12 months. Find out what the LOTUS study reveals for patients and doctors.
Neuren Pharmaceuticals advances NNZ-2591 for Phelan-McDermid syndrome to Phase 3 clinical trial
Neuren Pharmaceuticals has secured approval from the United States Food and Drug Administration (FDA) to progress into the…
Astellas Pharma to acquire stake in biotech company Taysha GeneTherapies
Astellas Pharma has signed an agreement to acquire a 15% stake in US-based biotech company Taysha GeneTherapies through…
LAVENDER phase 3 clinical trial launched for trofinetide in Rett syndrome by Acadia Pharmaceuticals
US biopharma company Acadia Pharmaceuticals has launched the LAVENDER phase 3 clinical trial to evaluate the efficacy and…