Our Accomplishments

Every dollar donated to Cure MDS has been donated or raised by families with an afflicted loved one. This funding has resulted in critically important advances in research on MDS.

• Since 2010 MDS families and their networks have raised $5 million, all of which supports ongoing research

• Funded the crucial experiment in the lab of Huda Zoghbi that determined that MDS is reversible

• Based on the reversibility results, currently providing funding to the Zoghbi lab to develop an antisense oligonucleotide therapeutic to treat MDS

• Funded two additional potentially curative projects, one in the lab of Ronald Cohn and the other in the lab of Anastasia Khovorova

• In partnership with the Blake McMillan Trust and the Van Wright Foundation provided support to the MECP2 Duplication Syndrome Database carried out by Dr. Helen Leonard.

MECP2 Duplication Syndrome is REVERSIBLE!

The MECP2 gene is also involved in Rett Syndrome, a genetic disorder that has a well-established scientific community. The Rett Syndrome Research Trust (RSRT) is the world’s largest funder of Rett research having invested over $66 million since 2008. In an effort to leverage RSRT’s knowledge base and global scientific networks, parents of MDS children inquired in 2010 whether the organization could also administer research efforts for MDS. a concerted, parent-driven effort was established to raise funds for the initial MECP2 reversal experiments conducted at the Baylor College of Medicine. Each MECP2 family pledged to raise $401.00 in funds to cover the research program cost. The initiative was a success, and the 401 Project was born. To date, the collective efforts of dedicated families has raised over 4 million dollars.

10 years on, the scientific community has taken notice and we are proud to have funded eight research projects. the 401 project has served its purpose. Our ongoing, collective effort is manifest in our name: Cure MECP2 Duplication Syndrome.

Importantly, this human capital comes at no cost to Cure MDS. 100% of every dollar contributed is invested in research – not a single penny goes to overhead.

Although Rett Syndrome has more awareness, more families, and more money, the fact is that reducing protein levels for MDS is technologically easier than boosting protein levels for Rett. In that respect MDS should be easier to cure/treat than Rett Syndrome.