Over the past couple of months we have been holding our breaths as results from the FDA trail of a drug formerly known as NNZ-2566 were coming out. This is the drug closest to market for Rett Syndrome and would be the first ever treatment of the actual disease. Over the weekend, the FDA denied Breakthrough Therapy Status.
That doesn’t mean it doesn’t work. It doesn’t mean the trials will stop. It’s just bureaucratic designations. You see it’s impossible to have standardized testing for Rett Syndrome because the apraxia is so severe. And since the results were all based on clinical observation, it was impossible for the FDA to say beyond a shadow a doubt that this treatment deserved the distinction. At least that’s my understanding of the situation. If you read here from Rett Syndrome’s website you’ll get the official position on it – https://www.rettsyndrome.org/blog/neuren-breakthrough-status-recent-update
Or check out this video from RSRT: https://www.youtube.com/watch?v=udUvAgN04Qc
It’s not a step backward at all – it’s just not as large a step forward. Either way, it’s a step in the right direction.