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…
A recent article from SciTechDaily describes the promising approach instituted at a private, non-profit biomedical research firm in San Francisco that incorporates data science, AI, stem cell biology, CRISPR, and…
Editor's Note: Patient Worthy is honored to share this submission from Katie DiLorenzo, SVP, Patient and Pharmacy Services, PANTHERx® Rare. If you’re living with a rare disease or caring for…
WebMD published a story about a young woman, Maddie, who had spent nine years in wheelchair confinement resulting from a rare metabolic disease related to kidney stones. As her doctor…
Most people see the bed first. That’s what defines me to them. The bed in the living room, the fact that I’m always in it. When they picture me, they…
For nearly two decades, I’ve been building and maintaining a global online community for individuals and families affected by Moebius syndrome, a rare neurological condition that most people have never…
I have battled several autoimmune and neurological conditions that are triggered by food and other factors for over 50 years. It took most of my life to reach not one,…
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