ELEVIDYS ENHANCE Study: A Phase 4 Trial Begins to Evaluate Safety and Effectiveness in Duchenne Muscular Dystrophy
The ENHANCE study is a Phase 4 clinical trial designed to evaluate the real-world safety and effectiveness of ELEVIDYS in patients with Duchenne muscular...
Medera’s Gene Therapy for DMD Cardiomyopathy Gains Momentum with FDA Fast Track Designation
A promising advancement in gene therapy for DMD cardiomyopathy has emerged as Medera Inc. receives Fast Track designation from the U.S. Food and Drug...
SGT-003 Gene Therapy: INSPIRE DUCHENNE Phase 3 Study Begins
Solid Biosciences has reported encouraging interim results from its ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial, evaluating the investigational gene therapy SGT-003 for Duchenne...
Atossa Presented New (Z)-Endoxifen Research Showing Improved Muscle Function in Duchenne Mouse Model
At the 2026 MDA Clinical & Scientific Conference in Orlando, researchers from Atossa Therapeutics presented new preclinical data about (Z)-endoxifen and its potential use...
Del-Zota Phase 3 SAFARI44 Clinical Trial for Duchenne Muscular Dystrophy: Study Design, Data, and Clinical Significance
The del-zota phase 3 SAFARI44 clinical trial for Duchenne muscular dystrophy represents a major step toward developing targeted treatments for patients with exon-44–amenable genetic...