RARE Daily

Rare Leader: Tiffany House, President Acid Maltase Deficiency Association

May 6, 2021

The Basics
Name:  Tiffany House

Title: President

Organization:  Acid Maltase Deficiency Association (AMDA)

Social Media Links:

Disease focus
: Acid Maltase Deficiency or Pompe disease is caused by a complete or partial deficiency of the lysosomal enzyme, alpha-glucosidase. This enzyme is necessary to break down glycogen and to convert it into glucose. Without this enzyme, glycogen, a thick sticky substance, accumulates in the lysosomes (sacs within the muscle cells) and leads to severe muscle degradation. It predominately affects the heart, skeletal, and respiratory muscles of the patient.

Headquarters: San Antonio, Texas

How did you become involved in rare disease: I have Pompe disease, and when I was diagnosed in 1995 there wasn’t an organization focused on just Pompe disease in the United States. To fill that void, my parents started the AMDA to focus on research and raising awareness for Pompe.

Education: B.A. and M.A. in English from the University of Texas, San Antonio and a J.D. from St Mary’s University Law School.

The Organization
Organization’s mandate: We’re focused on raising awareness of Pompe disease. More importantly than that, we focus on promoting research into Pompe and supporting patients and their families with Pompe.

Organization’s strategy: We have a variety of ways that we support patients. We do outreach through our website and the different Facebook groups that are there for Pompe. We also do a lot of one-on-one support of patients. If a patient or their family is facing a specific issue, whether it is trying to get a diagnosis, needing help finding a doctor, or just have questions, we provide support either through our patient coordinator (who is a registered nurse), or we connect them with other patients in the community that can support them. When it comes to research, we provide an annual research grant that is supported by an annual fundraiser. For the grant, we put out a call for applications and then through a committee process, choose a project that we feel will be most beneficial to the community. Due to COVID, we unfortunately were not able to hold a fundraiser last year because it’s an in-person fundraiser. However, we’re excited to hold one this year on August 28 in San Antonio, Texas.

Funding strategy: We reach out to industry and private donors for our organizational funding and educational funding. For the research grant we have an annual fundraiser (as mentioned above).

What’s changing at your organization in the next year: We recently relaunched our website after quite a few years. With that up and running, we are hoping to add new content, especially material geared to patients diagnosed by newborn screening. What we’re finding is infantile onset patients who are diagnosed early, that’s a pretty quick rush to get on treatment; that’s one component. For the late onset patients who are being picked up, we’re finding that they need a different level of support. There are a lot of questions they have about what “late” onset means in their case. Does it mean symptoms at 1-year-old or symptoms at 70-years-old? Through talking with these families we are hoping to come to some understanding of what support is most needed at that level, and how to best provide it.

Management Style
Management philosophy: I’ve been labeled a consensus builder. I try to seek out a variety of opinions and take the best pieces from everything that I hear. From there, I go in a direction that I feel will be best. At the same time, I understand that if it’s not, it’s okay to shift direction.

Guiding principles for running an effective organization: I’d say the same answer. Trying to bring in as many voices as I can. I’m addressing the needs of the entire community, and with Pompe we have a very diverse disease spectrum. So, I try to be open to feedback from everyone and to incorporate as much as possible. On the other hand, I have to accept that I’m not going to always make everybody happy and I’m not going to always please everybody. So, just doing the best that I can.

Best way to keep your organization relevant: Continuing to listen to your community.

Why people like working with you:  You’d have to ask people who worked for me. I don’t know how to answer that one.

Mentor: I learned a lot from watching my parents run the organization in the early days. I’m also fortunate to have some very good friends around the world who run their own Pompe organizations. I’ve learned from them how to look at the big picture, how to adapt, how to keep going even when it gets hard. It can be very hard at times, but also very rewarding.

On the Job
What inspires you: The patients we help—hearing that some of what we provide has been helpful to them and helps them get through some of their toughest days.

What makes you hopeful: In the Pompe community we are fortunate to have a fantastic group of researchers and medical professionals who have come together and are part of our community. To see the new research that is coming out and where things are likely to be heading, even if it’s 10 or 15 years away, makes me hopeful.

Best organization decision: One of the best decisions I’ve made is learning to ask for help and bringing in additional staff.

Hardest lesson learned: That I need to learn to ask for help. And, that you can’t always please everybody.

Toughest organization decision: I don’t think there have been hard decisions because it’s always been about doing what is best for the community. That’s not a hard decision to make.

Biggest missed opportunity: Because we are a smaller organization it’s always hard to be involved in every activity we want to be involved in. And then, with my own health, I’m not able to attend as many meetings in person as I would like to attend. I don’t see one huge opportunity that we’ve missed. I see small, missed opportunities.

Like best about the job: I’m helping people and believe what I am doing is important and necessary.

Like least about the job: That there’s never enough time in the day to do what I want to do.

Pet peeve: In terms of the organization, some of the patients don’t understand the history of where we’ve come from in terms of treatment history and organizational activities. I think it’s important to know where we’ve come from so you know where we are going and where we should be going.

First choice for a new career: First choice would be to more directly utilize my law degree.

Personal Taste
Most influential book: I’m an avid reader. Historical fiction is my favorite genre.

Favorite movie: My Big Fat Greek Wedding

Favorite music
: Country

Favorite food: French fries

Guilty pleasure: Law and Order marathons

Favorite way to spend free time: With my nieces and nephews.

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