Rett

Not sure if these have been posted before.  Nothing much new except for the comments below:

Phase 2 Trial of NNZ-2566 Shows Activity in Rett Syndrome
Christin Melton
Published Online: Wednesday, Dec 17, 2014

http://www.raredr.com/articles/Phase-2-NNZ-2566-Rett-Syndrome

Dr Alan Percy, Professor of Pediatrics, Neurology, Neurobiology, Genetics, and Psychology at the University of Alabama in Birmingham, was an investigator for the study. In an interview with Rare Disease Report, he said:

"We believe that this short-term treatment provided evidence of improved communication, awareness, and motor performance that was supported by an improvement in quality of life [as reported] by the parents."

He cautioned that although the results are encouraging, "It is essential to conduct longer term studies in adults as well as conduct similar studies in younger individuals." Dr Percy said the investigators hope to begin additional studies in the coming year. They expect to publish results of the completed trial early in 2015.



Phase 2 Rett Syndrome Study Shows Promise
James Radke
Published Online: Wednesday, Nov 12, 2014

http://www.raredr.com/articles/Phase-2-Rett-Syndrome-Study-Shows-Promise

Clinical investigator, Alan Percy MD, Professor of Pediatric Neurology at the University of Alabama stated:

“The results of this trial suggest a very promising proof of concept as we continue on the pathway to develop a disease-altering treatment for girls and women with Rett syndrome. Not only was this short-term trial managed successfully, but also the data analyses were conducted in a very robust fashion.”