mRNA Studies in DMD Treatment: Future Therapeutic Candidates
Given that FDA-approved exon skipping therapies and gene therapy for Duchenne Muscular Dystrophy, which are known to not produce sufficient levels of dystrophin and...
Miraculous Steroid Vamorolone (Agamree) Could Offer Safer Treatment for DMD
A novel steroid medication that is used to treat Duchenne muscular dystrophy has showed great potential in treating inflammatory conditions including rheumatoid arthritis, according...
Keros Therapeutics Announces Initial Topline Results from the Phase 1 Clinical Trial of KER-065 in Healthy Volunteers
Keros Therapeutics, a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are...
Upcoming Exon 51 Skipping Therapies for the Treatment of Duchenne Muscular Dystrophy
Duchenne Exon 51 skipping therapies are innovative treatments aimed at addressing the genetic mutation responsible for Duchenne muscular dystrophy (DMD). By skipping exon 51...
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 patients treated with exon 51...