On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological…
This is Part 3 of three articles I wrote about pain. The first 2 are entitled: Part 1- The real impact of pain that people cannot see Part 2- Life…
My journey with cardiac amyloidosis started shortly after I retired as a firefighter/paramedic in spring 2018. I started with trigger fingers—one in 2018 and two more in 2020. Also in…
While surgery has long been the standard for removing solid tumors, a revolutionary technique called histotripsy is offering an alternative, as reported on MedPage Today. Histotripsy uses high-intensity, focused ultrasound…
Remix Therapeutics has achieved a significant regulatory milestone with the U.S. Food and Drug Administration granting Fast Track designation to REM-422, a first-in-class small molecule therapy designed to treat recurrent,…
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 years it’s because of everything…
On 12/12/2025, I was admitted into the ER for a thrombectomy due to a bilateral pulmonary embolism. While I am overweight and sometimes eat like a raccoon in the trash,…
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 idiopathic hypersomnia (IH), sleep disorders…
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 embodies our mission of providing…
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: what will last beyond the…
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 family members, researchers, and specialists.…
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 Hunter syndrome, marking a pivotal…
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), for chronic weight management. The…
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 2023, I assumed it was…
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 States for treating cholestatic pruritus…
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 heart murmur. At the time,…
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 recently published in Science Advance.…
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 4.45 mg and 17.8 mg.…
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 Foundation). To see this article…
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 the community. We fight for…
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 was a decade in the…
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 we adopted in 2013—now teenagers…
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 gain. The EMANATE trial, conducted…
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 broadly across cancer care. The…
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 worthy or any affiliated organization,…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
