Author Archives: Forest Ray PhD

Stem Cell Therapy VTA-110 Named FDA Orphan Drug for DMD Treatment

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vita Therapeutics‘ VTA-110, a candidate therapy for people with Duchenne muscular dystrophy (DMD). The designation grants various development incentives to medications that address rare disorders affecting fewer than 200,000 people annually in the…

Benitec Begins Early Study of BB-301 Gene Therapy for OPMD

Benitec Biopharma has begun the first study to support the start of clinical trials of BB-301, a potential gene therapy for people with oculopharyngeal muscular dystrophy (OPMD). The BB-301 Tissue Transduction Study is the first of three studies to be conducted in large animals, with the overall…

NS Pharma Opens Support Hub to Help DMD Patients Access Treatments

NS Pharma has launched a support hub, called NS Support, for people in the U.S. with Duchenne muscular dystrophy (DMD) and their healthcare providers. NS Support will provide patients and clinicians with information on product availability and program enrollment. The hub can be reached by telephone at 833-677-8778, Monday…

FDA Asked to Approve Casimersen, Exon 45 Skipping DMD Therapy

Sarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) seeking approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 45 skipping. “The completion of our casimersen submission is an important milestone in our journey to advance treatments…