Patient Perspectives

Rare Village Foundation Lets Rare Disease Families Know They're Not Alone

Devin Rose | October 05, 2023

“When you get that diagnosis, your life is forever changed, "said Kasey Woleben about receiving the news that her son, Will, had a rare genetic disease. Woleben (on the far right, below, with Will) is co-founder of a non-profit organization that serves as a fundraising platform for families like her own.

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From Camp to Capitol Hill, Jordan Howard Advocates for Hemophilia

Devin Rose | April 17, 2023

College freshman Jordan Howard, who lives with severe hemophilia B, recently spent his first Rare Disease Week in Washington, D.C. talking with legislators about the benefits of newborn screening.

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Through the Pain of Fabry Disease, Artist Wes Burian Hasn’t Let Go of Hope

Devin Rose | February 28, 2023

In recognition of Rare Disease Day, artist and clinical trial participant Wes Burian shared his story about living life with Fabry disease, a lysosomal storage disorder.

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Meet Amber Freed, Mother and SLC6A1 Advocate

ASGCT Staff | January 28, 2021

When he was 18 months old, Amber Freed's son, Maxwell, was diagnosed with SLC6A1, a rare genetic disorder that causes seizures, severe movement and speech disorders, and intellectual disability.

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Sean Doerr Shares How He's Embracing Life with Pompe Disease

ASGCT Staff | February 06, 2020

In this Q&A, Sean Doerr, 29, talks about living his life with a genetic disorder that took years to diagnose, the importance of being your own advocate, and why he believes it's an "exciting time to have Pompe disease."

Education  |  Patient Perspectives
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Watch "Life After Gene Therapy" from ASGCT and NORD

ASGCT Staff | January 10, 2020

The fifth and final webinar in this series explores the personal journeys  of two patients who received gene therapy.

Education  |  Patient Perspectives
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2024

Register for the ASGCT Policy Summit

September 23-24, 2024 | Washington, D.C.

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