nnz-2566 retts trial , page-7

abitlikeme, I sincerely hope a breakthrough is just around the corner for your daughter. Dr Larry Glass and his team are very optimistic..

I'm sure that all posters here would jump for joy if we saw a reversal, not only for a return on investment, but for the happiness it would bring to your family. Reading your post shows just how important funding is to expedite trials, etc.

I wish you and your family the best. I'm heavily invested in NEU and I'd honestly lose the lot to know that one child was cured.

Below shows some HOPE. Good luck and best wishes.

The challenge of developing therapies for MECP2 disorders[42]
Recent studies, funded by the International Rett Syndrome Foundation, demonstrate that neurological deficits resulting from loss of MECP2 can be reversed upon restoration of gene function. These studies are quite exciting because they show that neurons that have suffered the consequences of loss of MECP2 function are poised to regain functionality once MECP2 is provided gradually and in the correct spatial distribution. This provides hope for restoring neuronal function in patients with RTT. However, the strategy in humans will require providing the critical factors that function downstream of MECP2 because of the challenges in delivering the correct MECP2 dosage only to neurons that lack it, given that the slightest perturbation in MECP2 level is deleterious. Thus, therapeutic strategies necessitate the identification of the molecular mechanisms underlying individual RTT phenotypes and picking out the candidates that can be therapeutically targeted. The next phase of research needs to assess how complete the recovery is. Clearly, lethality, level of activity, and hippocampal plasticity are rescued, but are the animals free of any other RTT signs such as social behavior deficits, anxiety, and cognitive impairments? Since postnatal rescue results in viability, it will be important to evaluate if even the subtler phenotypes of RTT and MECP2 disorders are rescued when protein function is restored postnatally. This is particularly important given emerging data about early neonatal experiences and their long-term effects on behavior in adults.