Research

Precision BioSciences Announced the Start of Phase 1/​2a (FUNCTION-DMD) Study of PBGENE-DMD in Ambulatory Patients with Duchenne

Precision BioSciences announced the commencement of a Phase 1/​2a trial of PBGENE-DMD (NCT07429240) in ambulatory patients with Duchenne aged 2 to 7 years. In...

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...

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