IndoUSrare and GORD Foundation are honored to announce the 2026 Abbey Meyers Khushi Bridging RARE Award Honorees: Rare Disease is a global problem, a human problem, and the individuals below…
Editor's Note: Patient Worthy is honored to present this story, shared with us by our friends at the Courageous Parents Network. Originally written by Dr. Adebimpe Adewusi, the article can…
Editor's Note: Patient Worthy is honored to share this essay from Carrie Ostrea. I have been fortunate to share my rare disease daughter's story for seventeen years. I have shared…
At 22, a young person's world should be filled with possibilities—a career to build, places to travel, friendships to make, and a future to imagine. Instead, this young man, David,…
Editor's Note: Patient Worthy is honored to share the following article, originally published by our friends at CURE SYNGAP1. To see the article in its original format, please click here.…
As reported on PR NewsWire, Orsini has been named a specialty pharmacy partner for Priovant’s LISRAYA™ (brepocitinib), a once-daily oral treatment recently approved by the US Food and Drug Administration…
A Rare Disease Gets Its First FDA-Approved Medicine—And It Works Differently Than Anything Available Before Imagine your immune system getting the signals completely wrong. Instead of protecting you from infection,…
Editor's Note: Patient Worthy is honored to share this story, submitted to us by Richard Poulin III. Late one night, a seemingly ordinary Facebook post changed the course of our…
As reported on Newsweek, a novel experimental treatment for Guillain-Barré syndrome (GBS) may represent a major advance for patients living with the rare neurological disorder. New findings released by biotechnology…
The Basic Problem: Why One Bad Gene Can Cause Big Trouble Imagine your body as a giant factory. Like any factory, it needs quality control—machines that make sure good products…
As reported on BioSpace, the U.S. Food and Drug Administration has approved Regeneron’s Pasatru (garetosmab) for adults with fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder that causes bone to…
Reflections on V122I, Genetic Risk, and the Questions Black Families Are Still Asking Editor's Note: This article was written and submitted to us by Erin Poyant of #hattrnextgen®. "When you…
Editor's Note: This article was shared with us by Jo Kaur. To see it in its original format, please click here. Riaan Singh Digeorge, age 6, of New York…
As reported on MedIndia, England is set to introduce nationwide newborn screening for spinal muscular atrophy (SMA) in October 2027, a move designed to accelerate diagnosis and enable earlier treatment…
Last year, I came down with a severe autoimmune disorder. It came out of nowhere. And it was bad. I was hospitalized three times over the course of a few…
Grifols, as announced in a recent press release, has officially launched FESILTY™ (fibrinogen, human-chmt) in the United States. The therapy is approved to treat sudden, severe bleeding in adults and…
As reported on Scientific American, a growing body of research suggests that Alzheimer’s disease progression is driven by a complex interplay between beta-amyloid plaques and tau neurofibrillary tangles. While both…
Recently at the World Orphan Drug Conference in Boston, Patient Worthy had the opportunity to meet with Dave Pearce PhD, Chair of International Rare Disease Research Consortium, (IRDiRC) Professor of…
In a recent statement, CONNECTA Therapeutics, a biotechnology company focused on disorders of the central nervous system, has announced the start of its Phase IIa clinical trial for CTH120, a…
As reported on Business Wire, TearSolutions, a clinical-stage biotechnology company focused on ophthalmic therapies, has announced key regulatory and clinical progress for its investigational drug Lacripep® in the treatment of…
Website: https://fabrynetwork.org/youngadults Instagram Young Adults: @fabryyoungadults Instagram FIN: @fabryintnetwork 11 June 2026 - FIN launches international Young Adults Guidebook created by and for young adults living with Fabry disease. Young…
The human body is governed by an ancient, rapid-response defense system. When an invader attacks, the innate immune system rushes to the front lines like firefighters to a blaze. But…
Editor's Note: Patient Worthy is honored to present this story, shared with us by our friends at The Mast Cell Disease Society. To see the article in its original format,…
Editor's Note: This article was originally written and shared with us by Caleigh Haber. This day hits really close to home for me. I was born with Cystic Fibrosis in…
As reported on MedicalXpress, a newly developed RNA-focused analytical method may improve the detection and characterization of repeat expansion disorders, a group of genetic conditions that includes Huntington’s disease, amyotrophic…
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