The FDA Designates IPS HEART’s GIVI-MPC Stem Cell Therapy for Becker Muscular Dystrophy an Orphan Drug
The FDA has granted IPS HEART Orphan Drug Designation (ODD) for GIVI-MPCs because of its exceptional capacity to produce new muscle with full length...
ImmunoForge Receives FDA IND Approval for Phase 2 Clinical Trial of ‘Pemziviptadil’ for DMD Cardiomyopathy
A medication for DMD cardiomyopathy, pemziviptadil is used once a week and is based on ImmunForge's ELP Platform (Elastin Like Polypeptide Platform), a long-acting...
Nippon Shinyaku Releases First Clinical Trial (First in Human) of Brogidirsen NS-089/NCNP-02 for the Treatment of Duchenne
The National Center of Neurology and Psychiatry is announced the publication of a research paper in Cell Reports Medicine detailing the results of an...
What is Ataluren (Translarna)? What is Ataluren Used For?
Ataluren (Translarna) is an investigational drug used to delay disease progression in outpatients with Duchenne muscular dystrophy (DMD), a progressive and fatal neuromuscular disease....
Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy
The results of new gene therapy studies for Duchenne muscular dystrophy disease will be watched by curious eyes in 2025. The trial phases of...