Posted on 11/05/202111/05/2021Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
Posted on 29/04/202129/04/2021Adverum Biotechnologies announced a Suspected Unexpected Serious Adverse Reaction (SUSAR) of hypotony (clinically-relevant decrease in ocular pressure) in its INFINITY clinical trial evaluating ADVM-022 gene therapy for the treatment of diabetic macular edema
Posted on 29/03/202129/03/2021Independent investigation shows that etranacogene dezaparvovec is highly unlikely to be the cause of HCC in HOPE-B pivotal trial
Posted on 29/03/202129/03/2021The U.S. Food and Drug Administration approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to, or whose disease has returned after, at least four prior lines (different types) of therapy
Posted on 26/03/202126/03/2021ProQR Announces Positive Results from Clinical Trial of QR-421a in Usher Syndrome and Plans to Start Pivotal Trials
