Honoring Advocacy: Celebrating Tiffany House and the RareVoice Awards
This week is Rare Disease Week, with events and activities taking place worldwide to bring awareness to rare diseases and honor the community. One such event is “Rare Disease Week on Capitol Hill,” where Tiffany House is being featured as a RareVoice Awardee for State Advocacy — Patient Advocate or Organization. Her recognitions during Rare Disease Week has reminded us at the AMDA of the importance of advocacy, sharing stories, and letting your voice be heard.
We all knew that Tiffany never sought accolades or the spotlight for herself. Instead, she consistently redirected attention toward raising awareness of Pompe disease (specifically, newborn screening and early detection and diagnosis) advocating for the rare disease community, and highlighting the urgent needs of patients and families. She often reminded us that the work is far from over and that we must continue striving for better treatment options, improved disease management, and a deeper understanding of Pompe.
Advocacy is More Than an Effort — It is a Passion That Requires True Commitment
Advocacy takes time, and it doesn’t stop — especially when meaningful change involves policy and laws that must be carefully considered, developed, and implemented. It is driven not only by urgency, but by persistence, patience, and unwavering dedication.
The progress made in newborn screening for Pompe disease is a testament to that commitment:
- After years of advocacy, Pompe disease was officially added to the US Recommended Uniform Screening Panel (RUSP) for newborn screening in 2015.
- On August 18, 2025, Texas DSHS launched newborn screening for Pompe, MPS I, MPS II, and Krabbe.
- As of August 2025, 47 states plus D.C. screened for Pompe disease, with Texas being the most recent to add it. The AMDA’s advocacy efforts will continue until all 50 states add Pompe disease to their newborn screening panels to include RUSP alignment.
Your Voice Matters
When you use your voice to engage in legislation, share your story, and speak up for what needs to change, you help shape decisions that affect real lives. Lawmakers and leaders rely on the lived experience of individuals to understand the true impact of policies beyond the page.
Sharing your story brings humanity to legislation, turning abstract issues into real, urgent priorities. It builds awareness, fosters empathy, and inspires action. Change doesn’t happen overnight: It happens when people commit to using their voices consistently, courageously, and with purpose.
Every story shared and every voice raised is a step toward meaningful, positive change.
Advice for People Wanting to Make a Change
If you want to make change, start by believing that your voice matters. Share your story — your lived experience has the power to educate, humanize issues, and move others to action in ways facts and statistics alone cannot. Learn about the policies and legislation connected to your cause so you can speak with clarity and purpose, and build relationships with others who share your commitment.
Meaningful change often involves complex systems, policies, and laws, and progress may feel slow. It requires true commitment, persistence, and patience. There will be challenges, and not every effort will lead to immediate results, but every conversation, story shared, and step forward helps build momentum.
Above all else, don’t get discouraged. Keep showing up. Keep speaking out. Keep fighting for what you believe in. Change happens because people refuse to give up; your dedication can make a lasting difference. That’s what Tiffany championed. Her championing continues to guide the AMDA in its actions now that she is no longer here.
Resources for Advocates: The EveryLife Foundation and NORD
If you are ready to take action, you don’t have to start from scratch. Two organizations in particular offer powerful resources to help people diagnosed with a rare disease, caregivers, and allies find their footing as advocates and amplify their voices on a national stage.
EveryLife Foundation for Rare Diseases
The EveryLife Foundation for Rare Diseases is a nonprofit, nonpartisan organization dedicated to empowering the rare disease community to advocate for science-driven legislation and policy. Importantly, the EveryLife Foundation doesn’t speak for patients, it provides the training, education, resources, and opportunities to help patients make their own voices heard. Through its Rare Disease Legislative Advocates (RDLA) program, the Foundation offers free grassroots advocacy tools, including action alerts, monthly webinars, legislative scorecards, and events open to patients and caregivers at no cost. It also coordinates Rare Disease Week on Capitol Hill — the very event where Tiffany House was honored — bringing advocates from across the country together to share their stories with legislators in Washington, D.C.
National Organization for Rare Disorders
The National Organization for Rare Disorders (NORD) is the leading independent patient advocacy organization representing individuals and families affected by rare diseases in the United States. NORD offers a comprehensive Rare Disease Database with information on more than 1,200 conditions, patient and caregiver assistance programs, and a network of Rare Disease Centers of Excellence. NORD’s Rare Action Network® connects advocates with tools, training, and resources to drive change at both the national and state levels. NORD is also the official US sponsor of Rare Disease Day, held each year on the last day of February. This serves as a reminder that the rare disease community stands together in its call for better access to care, research funding, and improved policy.
Both organizations welcome new advocates; whether you are a patient, caregiver, clinician, or ally. Exploring their websites is a meaningful first step toward using your voice and joining a community that refuses to stop fighting for change.