Erythropoietic Protoporphyria
The Sun Sucks, But You Do Not
My whole life growing up, there was this specific buzzing sound that would come alive on the sunniest of days. I’ve been told over the
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Simple Daily Habits to Boost Your Whole-Body Health and Well-Being
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Bridging Nutrition Science and Patient Care: ANA and Jazz Pharmaceuticals Launch Cardiovascular Health Initiative
The American Nutrition Association (ANA) has announced a collaborative initiative with Jazz Pharmaceuticals designed to advance evidence-based nutritional education for individuals living with narcolepsy and
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The Mentor She Wished She Had – How Elizabeth Became a Lifeline for EB Families
This patient story is sponsored by Chiesi Global Rare Diseases and is promoted through the Patient Worthy Collaborative Content program. We only publish content that
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How to Build a Lasting Legacy While Living with Chronic Illness
Patients living with chronic or rare diseases and the caregivers coordinating appointments, medications, and daily life often carry a quiet question alongside the medical work:
Dravet Syndrome
The Dravet Syndrome Foundation of Spain’s Annual Meeting 2026
The Dravet Syndrome Foundation of Spain hosted its 2026 Annual Family Meeting on March 20 at the Rafaela Ybarra School in Madrid, bringing together roughly 100
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Breaking Through the Rare Disease Barrier: FDA’s Historic Approval of Denali’s Hunter Syndrome Treatment
The pharmaceutical industry celebrates a significant victory for rare disease patients as the Food and Drug Administration approved Denali Therapeutics’ groundbreaking enzyme replacement therapy for
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FDA clears higher‑dose Wegovy to support long‑term weight management
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has approved Novo Nordisk’s higher‑dose formulation of semaglutide, Wegovy HD (7.2 mg once weekly),
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I used to be really active: swimming, Tai Chi every week, practicing yoga. So, when I first felt persistent rib and back pain in late
Primary Biliary Cholangitis
Lynavoy Achieves FDA Approval: A Breakthrough Treatment for Cholestatic Pruritus in Primary Biliary Cholangitis
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Major Breakthrough: Discovery of a Pathway that Allows Brain Cells to Move Toxic Material in Huntington’s Disease
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Lupin Receives FDA Approval for Pitolisant: A Novel Solution for Narcolepsy Management
Lupin, a globally recognized pharmaceutical company, has achieved a significant regulatory milestone with the receipt of tentative FDA approval for Pitolisant tablets in strengths of
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Can Your Morning Coffee Really Help Prevent Type 2 Diabetes?
Editor’s Note: Patient Worthy is honored to present this article, shared with us by our friends at the Steatotic Liver Foundation (formerly the Fatty Liver
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Heather and Nathan’s Journey with Huntington’s Disease
Fight. I don’t think that those who aren’t in the Huntington’s Disease community understand what the word FIGHT truly means to those of us inside
Ehlers-Danlos Syndrome
My Journey with Ehlers-Danlos Syndrome
My name is Amanda. I was diagnosed with a genetic connective tissue disease called Hypermobile Ehlers-Danlos Syndrome (h-EDS) about four years ago. However, the diagnosis
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Faye’s Journey with Amyloidosis
My name is Faye. I’m blessed to be the wife of the love of my life, Brad, and the proud mom of two extraordinary boys
Obesity
Setmelanotide Shows Limited Efficacy in Heterozygous Obesity Gene Carriers
A major clinical trial has revealed disappointing results for setmelanotide, a promising obesity treatment, when used in patients carrying single genetic mutations associated with weight
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EveryLife Foundation for Rare DiseasesThe #RAREis Scholarship supports individuals with rare diseases in building their dreams. Beyond financial support, recipients are invited to participate in a mentorship program that connects them with peers who share similar experiences or areas of study.
Apply by April 28 at 2 PM ET at hubs.li/Q049_wpm0
WORLD ORPHAN DRUG CONFERENCEFrom June 9-11 in Boston, MA, the World Orphan Drug Congress USA will once again bring together pharmaceutical companies, government officials, patient advocates, and other crucial stakeholders who contribute to developing and improving access to life-saving therapies for rare disease patients. With over 280 speakers and 2,000 attendees, this event is an opportunity to develop innovative solutions to the challenges that orphan drug development poses.
Our Chief Mission Officer, Annie Kennedy, will speak on the first day of the event at 9:20 am during the keynote titled, “Building a Patient-Centered Policy Framework for Rare Disease Drug Development.” The keynote will focus on how policymakers, regulators, and industry can collaborate to create strategies that incorporate patient insights into all stages of drug development.
You can join Annie and the EveryLife Foundation at this event for free with a guest pass. Click the following link to access the guest pass application form to secure a spot: secure.terrapinn.com/V5/sponsor-guest/11031/a0AN200000inZjFMAU?utm_source=partners&utm_medium=eve...
You can learn more about this year’s World Orphan Drug Congress USA here: www.terrapinn.com/conference/world-orphan-drug-congress-usa/index.stm?utm_source=partners&utm_med...
