Shanghai Jiao Tong University School of Medicine Begins Clinical Trials of LE051 Exon 51 Skipping Therapy

The purpose of LE051 study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD...

Translarna (Ataluren) Rejected by EMA: Marketing Authorization Not Renewed

In May 2024, PTC Therapeutics had been announced that the European Commission (EC) has decided not to adopt the...

DMD WarrioR’s Turkish Representative Shares His Views on Elevidys Gene Therapy: Is It Effective and Why Is It Expensive?

The efficacy of Elevidys in treating Duchenne muscular dystrophy (DMD) has shown promising results in clinical studies, but are...