Side effects reported in GSK exon skipping trials

Side effects reported in GSK exon skipping trials

GlaxoSmithKline (GSK) has announced that several boys taking part in clinical trials of drisapersen (a molecular patch) have received hospital treatment for side effects including a reduced number of a type of blood cell and protein in the urine. We would like to reassure our supporters that we are working closely with GSK and that we remain confident that the safety of boys in the trial is of paramount importance and that all boys are being carefully monitored for side effects.

In a presentation in Rome last week, GlaxoSmithKline (GSK) announced that several boys in the trials of drispersen (a potential exone skipping drug) have experienced serious side effects. The company reported that a small number of boys in the trial had required hospital treatment for thrombocytopenia (a reduction in the number of cells called platelets which can lead to problems with blood clotting) or proteinuria (too much protein in the urine which can be a sign of kidney damage).

Since the presentation, we have contacted GSK to ask for more information. They have assured us that the safety of the boys in the clinical trials is of "paramount importance" and confirmed that all the boys taking part in the trial are being carefully monitored for any signs of side effects. The vast majority (about 96%) of boys who started the trial are still taking part and GSK is confident that their monitoring program will make sure that all boys participating in the trial are safe. They also said that any boy who shows signs of these side effects will be admitted to a hospital for treatment and recommended that "anyone participating in a drisapersen study that has questions or concerns should discuss these with their study investigator."