RARE Global Advocacy Alliance Member Spotlight: Melissa Chaikof

Melissa Chaikof

Organization Name
Usher 1F Collaborative

Disease Affiliation
Usher syndrome type 1F

Organization Mission
The mission of Usher 1F Collaborative is to fund medical research to find an effective treatment to save or restore the vision of those with Usher Syndrome type 1F.

What led you to the rare disease community?
I have two daughters with Usher syndrome type 1F, an ultrarare disease

What do you think are the areas that are lacking in the community (specific to your org or in general)?
Funding for research for a cure

What are some of the pain points?
Connecting with potential donors who don’t have a prior relationship to us or to Usher 1F

What are your areas of expertise?
Donor research, telling our story, managing our website, understanding the science to enough of an extent that I can explain it in lay terms.

Please describe any major milestones your organization has hit or has coming up that you are proud of.
Our 10th anniversary is this year – We took Usher 1F research from nothing to 9 labs working on a cure with $9M invested in that research. Most importantly, we have at least one gene therapy that is anticipated to be ready for testing in humans within 5 years.

What is your hope for the future for rare diseases?
Recognition that together we are not rare and that more NIH funding needs to be dedicated to rare disease research.