Searching for a cure, Improving lives
Prosensa is working towards long-term access to drisapersen Dear Patient Group Representative, As previously communicated, Prosensa received guidance from the United States Food and Drug Administration (FDA) on June 2, in which a regulatory pathway for accelerated approval was outlined for drisapersen. Based on this guidance, we communicated that we intend to submit a file to the
The SKIP-NMD research project started in November 2012 with the award of an EU grant. The project brought together a consortium of researchers, clinicians and companies from across the field to develop and test a new molecular patch for exon 53 of the dystrophin gene. Members of the SKIP-NMD consortium recently met in Rome for
Action Duchenne have teamed up with the Care and Trial Site Registry (CTSR), a registry that was established by TREAT-NMD to collect information about care settings and trial site infrastructure in neuromuscular centres around the world. If you work at a centre, kindly fill out (or update your answers to) the standard questions in the CTSR plus the extra page explicitly for Action Duchenne.
To access the Care and Trial Site Registry, please click below and sign in, if you are already registered. Otherwise, please sign up for a new account.
Please go through the questionnaire from top to bottom and answer all the questions (be sure to check the Disease Group ‘NMD’, or you will not see all the questions). The pages under ‘Clinical trial infrastructure’ need only be filled out if you are interested in participating in clinical trials.Fill in the survey