Connecting the Dots for a Community of Rare Disease Patients: Terri Klein, MPS Society

Terri Klein, CEO, MPS Society

Bio and Contact Info


00:00 How many patients are affected with MPS diseases?

04:42 What are the existing therapies?

08:34 Volunteering to be early members of clinical trials a selfless act

14:27 How do you raise awareness?

17:10 What makes a successful registry?

23:07 Evening the playing field

The MPS Society is a rare organization. Not just because it is an umbrella for the rare diseases that have in common lysosomal storage malfunction. But because it is one of the largest rare disease organizations in the world.

Terri Klein is the CEO leading the MPS Society. She says the group has over three thousand members on their roster. This brings some strength and resources in an area where low numbers intensifies an uphill battle.

Her job, she says, is to find those researchers out there working on the various MPS diseases--Hunter’s, Sanfillippo syndrome and about a dozen others--and make sure any and all science toward understanding, therapy and cures gets shared and developed. She "connects the dots" between research, funding, trial experts, parents and patients. Beyond the science and medicine, Terri says about half of her budget goes to care of patients.

So what is the state of existing therapies for these diseases now, and how close are we today to gene editing and gene therapy cures?

Terri has been instrumental in the creation of ConnectMPS, a very successful disease registry for MPS patients. What are her thoughts on how to make rare disease registers more impactful?