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,…
For people living with Lynch syndrome, the threat of cancer is a constant shadow. This inherited condition affects about one in 300 people, making them up to 80% more likely…
As reported on BioSpace, Moderna has entered a commercialization agreement with Recordati valued at up to $160 million to support late‑stage development and future market introduction of mRNA‑3927, an investigational…
As reported on Science Daily, a breast cancer vaccine tested more than 20 years ago is drawing renewed scientific interest after researchers discovered that every participant in the original trial…
As reported on Healio, glucagon-like peptide-1 (GLP-1)–based therapies are rapidly reshaping the landscape of diabetes and chronic kidney disease (CKD) management. Originally developed for glycemic control, these agents now play…
Immunoglobulin A nephropathy (IgAN) remains one of the most common primary glomerular diseases worldwide, yet effective treatments are limited. A new study, recently published in Frontiers, offers fresh evidence that…
Brussels — On February 24, 2026, the acclaimed Netflix documentary The Remarkable Life of Ibelin received the Media & Awareness Raising Award at the EURORDIS Black Pearl Awards. The honor…
As reported on PM Live, a new clinical trial hub focused on epidermolysis bullosa (EB) has been established at University Medical Center Groningen (UMCG) in the Netherlands through a collaboration…
To the patient, the parent, the caregiver, and the person in the "limbo" of waiting: I am one of you. My name is Aaron Jackson, and this is my story.…
As reported on MedPage Today, an oral targeted therapy may offer a safe and effective alternative to injectable treatment for children with achondroplasia, according to new phase II data published…
As reported by NewsWise, new clinical study led by investigators at the University of North Carolina at Chapel Hill is offering rare hope for families affected by Hunter syndrome, a…
CRISPR Therapeutics has announced encouraging early results for zugocaptagene geleucel (zugo-cel), its investigational allogeneic CAR T therapy targeting CD19, in both autoimmune diseases and hematologic malignancies. Autoimmune Disease Progress In…
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