The RettSyndrome.org bench-to-bedside path towards treatments and a “cure” for Rett Syndrome
Since Rett Syndrome is a potentially reversible neurological disorder, RettSyndrome.org is currently cultivating research programs that link basic, translational and clinical research. Part of the current strategy to treat and reverse Rett Syndrome is to focus on funding clinical research to re-purpose promising treatments that are already available or are poised to enter the clinic for other indications. In parallel, RettSyndrome.org proactively supports the development of novel “platform” technologies, mouse models and other strategies that facilitate or catalyze the translation of basic research out of the lab and into the clinic.
RettSyndrom.org's Research and Development Efforts
The path from basic research discovery to the creation of a new treatment is often a lengthy and expensive process. The average time for the development of a new central nervous system treatment is currently 15 years.
RettSyndrome.org seeks to accelerate the drug discovery and development process to identify and test new treatments for Rett Syndrome. The goal is to increase the movement of new treatments into the clinic through careful investment in promising early-stage drug discovery and development programs in academia and industry. Ultimately, the early-stage testing and validation of potential therapeutics can de-risk the later, more costly phases of drug development.
RettSyndrom.org: Aims to Bridge the “funding gap”
Academic investigators and early-stage biotechnology researchers often have difficulty finding support for novel, high risk drug discovery and development projects which are costly and can take many years to achieve their objectives. While Governmental and traditional foundations have generally favored the support of basic research programs, Pharmaceutical companies and venture capitalists tend to fund later-stage research and clinical trials that are further along in the drug development pipeline. Therefore, RettSyndrome.org seeks to fill a critical funding gap in translational research and aid scientists to enter the field of drug discovery for Rett Syndrome. Funding decisions are based on rigorous scientific peer-review by our Scientific Review Board, which consists of leading international researchers working on Rett Syndrome, drug discovery and other relevant disciplines. Through this process RettSyndrome.org provides thorough due diligence and proper validation of new research programs.
RettSyndrome.org is dedicated to helping develop better treatments to improve the quality of life of individuals with Rett Syndrome and ultimately find a “cure.” Treatment is defined as anything that improves the outcome of conditions caused by Rett Syndrome. In addition to therapeutics this may include assistive technologies, communication, physical therapy and education. RettSyndrome.org supports and facilitates global research into the causes, treatments and an eventual cure for Rett syndrome by:
- Promoting interdisciplinary cooperation
- Funding cutting edge basic, translational and clinical research
- Organizing research symposia and meetings
Research funding is currently focused in several mission-critical areas:
- Understanding the role of MeCP2 during normal brain development
- Characterizing the role of MeCP2, including MeCP2 target genes, in normal structure and function of the developing and adult nervous system
- Determination of the relationship between patterns of expression of MeCP2 and related proteins in the nervous system and the neurologic and behavioral phenotypes of patients with RTT and/or related animal models
- The investigation of neuronal abnormalities that result from MeCP2 dysfunction
- The development of novel assays and the testing of innovative therapeutic approaches to treat or reverse RTT
- Development of assistive technologies to improve the quality of life of individuals affected by RTT
- Support for advances in physical therapy, communication and education