Cincinnati, OH - October 12, 2015 - Rettsyndrome.org today announced that the Board of Directors has made a financial commitment, through the Research to Reality Campaign, to continue support for further clinical trials with trofinetide, a drug developed and manufactured by Neuren Pharmaceuticals.
Following a successful Phase 2 trial with trofinetide in adult women with Rett syndrome, and an encouraging meeting with the Food and Drug Administration, Neuren is moving forward with a Phase 2 tolerability trial for children with Rett syndrome. Concurrently, Neuren is working with the FDA to design a Phase 3 pivotal study with trofinetide as a treatment for Rett syndrome.
Rajat Shah, Chairman of Rettsyndrome.org's, commented "Rettsyndrome.org is proud to continue our partnership with Neuren. We are grateful for their work and commitment to the clinical trials of trofinetide. We are hopeful that these clinical trials will be successful."
There are no approved drug treatments for Rett, a devastating neurological disorder. These trial advancements are significant because they bring us closer to the first potential treatments, and the hope of improving quality of life for those suffering from Rett syndrome.
This is an optimistic time for the Rett syndrome community. Research efforts are rapidly advancing our understanding of the disorder and the first treatments are closer to a reality. Ultimately, it is hoped they will lead to a cure. Rettsyndrome.org's Research to Reality Campaign is ensuring that funding is available to advance promising clinical trials like those for trofinetide.
Rettsyndrome.org is committed to a full spectrum approach to Rett syndrome research by supporting clinical trials as well as basic, translational and neurohabilitation research to better understand, treat, and ultimately cure Rett syndrome.
Rettsyndrome.org, a 501(c)3 non-profit, is the most comprehensive organization dedicated to accelerating research of treatments and a cure for Rett syndrome and related disorders while providing information and resources to empower Rett families. As the world's leading private funder of Rett syndrome research, Rettsyndrome.org has funded over $34M in high-quality, peer-reviewed research grants and programs to date. The organization hosts the largest global gathering of Rett researchers and clinicians to establish research direction for the future. To learn more about our work and Rett syndrome, visit http://www.