Onward and Upward with Little Girl Steps!

Photo by  Scott Liddell

Photo by
Scott Liddell

From Rettland.org

Yesterday Rettsyndrome.org held a webinar with Steve Kaminsky on the important topic of participating in clinical trials. If you missed it here is a link to the audio. We will all move toward a cure faster when we are educated on the process of how we get there!


A very informative webinar! I encourage everyone to listen. Discussing multiple on-going or recently completed trials in various countries, Dr. Kaminsky answers many of the questions people have about trials, who is eligible, obstacles faced when deciding where they will occur and others.

About halfway through questions were asked about the probable next steps for Trofinetide (NNZ-2566). Paraphrasing- the probability of a Phase III in adults was nixed and the best news….a Phase II, similar to the one done in adults- low dose, high dose and placebo, in the pediatric population was probably the next step…AND if (puhleeze…WHEN) that was shown to be effective a Pivotal Phase III encompassing all the ages in both trials and multiple sites (hurray!) would be done. This is great news because it will make it easier to fill all the spaces of the trial!

Meanwhile, Dr. Kauffman is still recruiting for the IGF-1 trial in Boston.

The next seminar in the series will hopefully be with the researchers at Neuren! I’ll be tuning in for that one!

And, maybe the FDA will get it together before the end of the pediatric trial and grant Breakthrough Therapy Designation. Here’s hoping!

This entry was posted in Neuren Pharmaceuticals, rettland.org, rettsyndrome.org and tagged , , , , . Bookmark the permalink.

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