Functional Improvement Seen in Dyne’s One-Year DM1 Trial Data
One-year data from six adults with myotonic dystrophy type 1 (DM1) treated with zeleciment basivarsen at 6.8 mg/kg every...
One-year data from six adults with myotonic dystrophy type 1 (DM1) treated with zeleciment basivarsen at 6.8 mg/kg every...
Preclinical data from an optic nerve crush model showed that ExoPTEN, an exosome-delivered PTEN siRNA, produced a reproducible, dose-dependent...
Early interim data from Gain Therapeutics’ Phase 1b study of GT-02287 in Parkinson’s disease point to stability and improvements...
In updated Phase 1/2 data for unilateral mesial temporal lobe epilepsy (MTLE), a single low dose of NRTX-1001 produced...
Atea reported 98% SVR12 (210/215) in the per‑protocol, treatment‑adherent cohort and 95% SVR12 (245/259) in the per‑protocol, regardless‑of‑adherence cohort...
Biomed Valley Discoveries reported a 25% confirmed objective response rate (4 of 16 patients) with a median response duration...
ARV-102 delivered dose-dependent exposure in plasma and CSF, was well tolerated with no serious adverse events or discontinuations, and...
Topline data: In a prospective, multi-center IDE randomized controlled trial for chronic craniofacial pain, 69% of patients on active...
Quantum BioPharma has received final 90-day oral toxicity and toxicokinetic reports for Lucid-21-302 (Lucid-MS), positioning the program to support...
In a randomized, double-blind Phase 2 study of 77 patients with multiple system atrophy (MSA), ATH434 delivered a clinically...