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:
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
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
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),
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
GSK has announced a significant advancement in liver disease treatment with FDA approval of Lynavoy (linerixibat), marking the first medication specifically approved in the United
My diagnosis of rheumatic heart disease came on suddenly. I remember being 12, sitting at the doctor’s office, and was told that I have a
Researchers at Florida Atlantic University (FAU) have identified a cellular pathway that allows brain cells to share proteins and other toxic material. The study was
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
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
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
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
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
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
As reported on PharmaBiz, Illumina and Labcorp have announced an expanded strategic collaboration aimed at accelerating precision oncology by applying next-generation sequencing (NGS) technologies more
Editor’s Note: The views, thoughts, and opinions expressed in this article belong solely to the author and do not necessarily reflect the position of Patient
Immutep Limited has announced the discontinuation of TACTI-004, a pivotal Phase III clinical trial investigating eftilagimod alfa (efti) as a first-line treatment for advanced non-small
As reported on FiercePharma, China-based biotech Dizal Pharmaceutical has reported a pivotal phase 3 success for its oral EGFR inhibitor Zegfrovy (sunvozertinib), marking a potential
Understanding how SMA is tested is a key step toward earlier diagnosis and faster access to care. From genetic blood testing to newborn screening, modern tools are helping identify spinal muscular atrophy sooner and more accurately than ever before. #spinalmuscularatrophy #genetictesting #PatientWorthy
International Foundation for CDKL5 Research-IFCRHave you registered for the CDKL5 Family Conference yet? You won’t want to miss this.👇
We’re hosting a powerful, honest session on gene therapy - starting with real talk about where the science stands, what risks exist, and what expectations are realistic.
Then we’ll move into a community risk/benefit workshop where every voice matters. Whether you’re eager to join a trial, unsure, or not interested at all, we need your perspective to understand the full picture of our community’s hopes and concerns.
Your voice shapes the future - We hope to see you there!! 💜✨
#Conference2026 #GeneTherapy #EducationalFamilyConference #RareDiseaseCommunity #CDKL5 #IFCRCommunity
United Mitochondrial Disease FoundationLooking for connection and support? Join patients and families from the mito community at one of UMDF’s virtual Support Meetings. Visit our events calendar for login details and come be part of the conversation. umdf.org/events-calendar/
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