A brief history: During and since 2016, there has been frustration in this community regarding drug development.

Fast forward: PPMD strives for both accuracy and transparency in all information we release. We made the decision to edit and ultimately remove the Research Row: Gene Therapy – Current and Evolving Landscape panel recording in response to Sarepta’s security concern referenced in the July 29^th STAT article by Adam Feuerstein.

The question initially edited was similar to other questions and focused on the need to release data and the need for patients/parents to understand how they or their child responded to a given intervention in a timely manner, not years after the study ended. These data are critical for making decisions around what therapies to consider, how care changes, and what’s next for individual patients.

Further, the ‘totality of data’ of any given therapy needs to be understood, because, if approved, individuals and parents need to consider both benefit and risk of given therapies. This is especially important for all AAV-based therapies, as they are one-time therapies (at this time) and cannot be discontinued as in other therapies.

Next steps: PPMD will continue to encourage companies to develop policies for the release of clinical trial data across all trials. Data should be sent directly to the Principal Investigators no later than one year after the last participant completes the trial.

This is advocacy at its best – the power of families to apply pressure and demand change.