Today Roche announced that the first patient has entered the GENERATION HD1 study. A trial that will investigate the efficacy and safety of the huntingtin lowering drug RG6042 – formerly known as IONIS-HTTRx.
Over the last couple of years, the safety of the drug and short term effects, have been measured in 46 participants. Now longer-term safety and efficacy will be studied in more than 600 participants.