Dwayne’s Pompe story
Hi everyone, my name is Dwayne; I am 57 years old. I was diagnosed with Late Onset Pompe disease (LOPD) in November 2018 when I was 50 years old. I…
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Dwayne’s Pompe story
Our mission is to amplify the rare condition voice. One of the ways we achieve that is through collaborations across the community, from patients and caregivers to industry leaders in treatment research and development. To learn more, click here.
The Ruminations of a Grin2b Mom
Patient Worthy is grateful to present this article by way of the CureGRIN Foundation. CureGRIN is a foundation founded and run by parents who are committed to improving the lives of people…
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The Ruminations of a Grin2b Mom
Zoe Ridgeway: My BHD Journey
Patient Worthy is honored to be able to share Zoe Ridgeway's story by way of theBHDFoundation.org. Zoe's story was shared for Rare Disease Day 2025 and the BHD Foundation believes…
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Zoe Ridgeway: My BHD Journey
Levi’s Story with Jansen’s Metaphyseal Chondrodysplasia
Patient Worthy is honored to bring you Levi’s story courtesy of our partners Miracle Flights. Miracle Flights is a non-profit organization that provides pediatric patients and their families free commercial…
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Levi’s Story with Jansen’s Metaphyseal Chondrodysplasia
Katie’s Fight Against Bacterial Meningitis
Nothing about meningitis is fair. In mere moments it can devastate lives and destroy families. It can take away dreams and shatter plans. These factors make marking my 20th year…
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Katie’s Fight Against Bacterial Meningitis
Cervical Dystonia Changed My Career (But Not as You May Think)
"You have cervical dystonia", said my beloved family doctor of 20 years. He was almost exuberant, relieved to have finally uncovered the mystery of my severe neck pain, head tremors,…
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Cervical Dystonia Changed My Career (But Not as You May Think)
Jeremie Soriano – from Dwarf to Giant
For Part 1 of Jeremie's story, click here. I just want to share my rare disease story today! I used to be a theater actor and a performer on stage.…
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Jeremie Soriano – from Dwarf to Giant
Jeremie Soriano – An Acromegaly Story
I was diagnosed in 2021. It all started when I was hospitalized for about 3 weeks due to high blood glucose - Hyperglycemia. During my stay in the hospital, my…
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Jeremie Soriano – An Acromegaly Story
Good early progress but more docs need to engage, patients need to ask about Rezdiffra – Fatty Liver Foundation
Madrigal today released their first report of results following FDA approval of Rezdiffra for the treatment of NASH in April. Their results are on track according to company forecasts but…
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Good early progress but more docs need to engage, patients need to ask about Rezdiffra – Fatty Liver Foundation
Expanded Access: What Patients and Caregivers Need to Know
Imagine that your loved one is diagnosed with a rare disease. The first thing you do is to go to the internet to understand what you can about this disease…
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Expanded Access: What Patients and Caregivers Need to Know
Navigating the “Messy Middle” in Rare Pediatric Cancer Fundraising
By: Erin Santos-Primis, Mom to Isabella and Executive Director of the Isabella Santos Foundation Starting a nonprofit in honor of a loved one lost to a tragic disease is often…
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Navigating the “Messy Middle” in Rare Pediatric Cancer Fundraising
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Reducing the Itch: Life with Progressive Familial Intrahepatic Cholestasis (PFIC)
Acknowledgment: This patient story is sponsored by Ipsen Biopharmaceuticals, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
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Reducing the Itch: Life with Progressive Familial Intrahepatic Cholestasis (PFIC)
Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)
Acknowledgment: This story is sponsored by Neurocrine Biosciences and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…
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Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)
Connecting with the Community for Change
Acknowledgment: This story is sponsored by Otsuka America Pharmaceutical, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant,…
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Connecting with the Community for Change
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How to be a Patient Peer: Providing Resources for People with ADPKD
Acknowledgment: This story is sponsored by Otsuka America Pharmaceutical, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
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How to be a Patient Peer: Providing Resources for People with ADPKD
Shingles Patients Sharing Stories
Patient Worthy is working with a partner agency who is looking to engage with patients living with Shingles. If chosen, participants will have the chance to take part in a…
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Shingles Patients Sharing Stories
Patients with HAE – Reoccuring Bouts
Patient Worthy is working with a partner agency who is looking to engage with patients living with Hereditary Angioedema (HAE) who have reoccurring symptoms, more than 2 times a month.…
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Patients with HAE – Reoccuring Bouts
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Taking Charge of Your HAE Treatment to Help the Next Generation
HAE is not my story. But it’s definitely part of my story. Looking back, I realize that my late childhood and early teen years held some occasional, unexplained swelling events that…
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Taking Charge of Your HAE Treatment to Help the Next Generation
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Why Are Adults in the US Having Trouble Accessing Their Medications?
Modern medication has the ability to save lives and improve living standards. However, medicine is less beneficial when patients’ prescribed treatments are delayed, and entirely ineffective when patients forgo treatment…
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Why Are Adults in the US Having Trouble Accessing Their Medications?
5 Facts You May Not Know About Adult Growth Hormone Deficiency (AGHD)
Adult Growth Hormone Deficiency (AGHD) is a rare condition when your body produces less growth hormone (GH) than normal for your age and sex. AGHD may affect your body systems,…
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5 Facts You May Not Know About Adult Growth Hormone Deficiency (AGHD)
Teenager with Rare Disease Treks Across the Country with a Very Important Message
Gabriel Low, a 17-year-old from Hawaii, just completed a 3,000-mile bike ride across the country. He’s been riding since June 11th when he embarked from Seattle, WA to raise awareness,…
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Teenager with Rare Disease Treks Across the Country with a Very Important Message
