Rocket Pharmaceuticals Inc Danon Update Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Tyler Martin Van Buren - Cowen and Company, LLC, Research Division - Analyst : Thank you very much for the comprehensive and encouraging update. Three, I guess the first is -- maybe you could talk a little bit more about the time course of improvement from a wall thickness standpoint. It looks like they continue to improve beyond 12 months, which is nicely correlated with ejection fraction. And then two is, now that we have all this data, what functional and biomarker endpoints do you think might be best suited for a pivotal trial. The initial update last year seemed to focus on cardiac output. But again, the wall thickness, ejection fraction data seems pretty interesting. And then the third is just on the steroid induced myopathy, a quick follow-up. Can you talk a little bit more about the actual manifestation of it and how it affects these patients? I know you discussed how you plan to limit it in the future, but just wanted to hear a little bit more about that.

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Congrats on the update. First one is, is there a way you can quantify the normal fluctuation of LAMP2B and how that affects your interpretation of these and future data?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Got it. And then one on the pediatric cohort. How young would you want to go with the treatment? Is the goal to really hit that area where they're closer to disease progression where you could get the benefit of whatever durability there is? Or would you try and go as early in the disease after diagnosis is possible.

Question: Gil Joseph Blum - Needham & Company, LLC, Research Division - Analyst : Congrats on all the progress quick couple from us as well. Do you think that the progression of transplant may be considered as a functional outcome assessment in the longer term? And do you guys have any thoughts on the optimal intervention window that has before there's too much fibrosis in the heart. And is there potential patient-specific monitoring, which could assist in determining this window?