Ann: Phase IIb study of ATL1102 in DMD completes recruitment, page-172

It initially appeared that gene therapy was the great hope for the Duchenne community. Pfizer, however has exited stage left and there was a high degree of controversy stage right at the recent PPMD conference. The make-dystrophin-and-it will-be-better honeymoon appears to be over and for the first time in a good time, caregivers will be attentive to alternative treatments. Preliminary results by December from the ATL1102 Phase IIb study couldn't be better timed.

It appears that there was a heated exchange during a panel at the PPMD conference in relation to confirmatory trial data delays? Fat chance, however, in accessing the video of the panel event. What we are left with are comments from PPMD in a response to an article in STAT where they outlined that the 'totality of data' of any given therapy needs to be understood. Ouch. Somewhat pointedly, they go on to state that its especially important for all AAV-based therapies.

I was pleased to hear recent comments by our Chief Medical Advisor who, in an interview on JustStocks, said that its critical to incorporate the patient caregiver perspective in order to meet needs in a meaningful way. Building trust and enabling caregivers to make well informed decisions would appear to be the right way forward.

The two different approaches to caregiver concern are chalk and cheese. At the end of the day we are talking about real people facing immense challenges and hoping for a better life.