As reported on ScienceDaily, a research team at the Icahn School of Medicine at Mount Sinai has unveiled a novel immunotherapy that tackles metastatic cancer
Editor’s Note: We’re honored to share part 4 of 10 of an ongoing blog series, originally written by Elena Genik. When You Go to a
As reported on drugs.com, REGENXBIO has disclosed that the U.S. Food and Drug Administration has halted clinical testing of its investigational gene therapies RGX‑111 and
Editor’s Note: Patient Worthy is honored to share this patient story, provided to us by our friends at Heal Canada, and originally written for the
Roche has announced encouraging results from its Phase II trial of CT-388, an experimental obesity treatment that achieved placebo-adjusted weight loss of 22.5% over 48
The European Medicines Agency (EMA) has issued a positive opinion recommending that retifanlimab (Zynyz) be approved for an expanded indication to treat adults with advanced
Editor’s Note: Patient Worthy is honored to share part 3 in an ongoing 10-part series, written and shared with us by Elena Genik. When My
Researchers have developed an innovative deep learning model capable of accurately differentiating thyroid eye disease (TED) from orbital myositis using computed tomography (CT) imaging, potentially
As reported on Drugs.com, REGENXBIO has announced that the U.S. Food and Drug Administration (FDA) has halted clinical testing of its investigational gene therapies RGX‑111
Several highly esteemed Institutes such as the Mayo Clinic and CONICET have identified critical elements contributing to the aggressiveness of Pancreatic cancer. The survival rate
Moderna and Merck have announced promising five-year data for a groundbreaking personalized cancer treatment that combines mRNA technology with immunotherapy to prevent melanoma from returning
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
Editor’s Note: Patient Worthy is honored to share part 2 in an ongoing 10-part series, written and shared with us by Elena Genik. Before the
Cancer immunotherapy represents one of modern medicine’s greatest achievements, yet it’s also one of its most frustrating paradoxes: a treatment that works miracles for some
As reported on BioSpace, Moderna has entered a commercialization partnership with Italy‑based Recordati to support the development of mRNA‑3927, an investigational mRNA therapy for propionic
Osteosarcoma is a diagnosis no family should ever have to face. It is the most common primary bone cancer in children and adolescents, yet treatment
A groundbreaking genetic study published in The Journal of Clinical Endocrinology & Metabolism and reported by ScienceDaily.com has revealed that obesity and high blood pressure
Twenty years is a long time to believe a cancer chapter has closed. For two decades after my radical prostatectomy, I lived with the quiet
Meet Kathy! Living with ANCA-associated vasculitis for about 20 years, she shares her story in honor of Rare Disease Month to inspire others along their journey.
"I was an athlete in the pinnacle of my career after 25 years in the martial arts. I had a family business of a chain of martial arts schools and I was the CEO. It was a tough fight to get to where I was, and I lost my position in this business pretty quickly when I was diagnosed at age 50 with Microscopic Polyangiitis, which is an ANCA associated form of Vasculitis.
I was hospitalized for 21 days because I was in kidney failure. I found out the name of the disease through a kidney biopsy. I also found out there was no cure, and it was a Rare disease with fewer than 50,000 cases in the United States. I was lucky to find that the leading expert in this disease worked at a University only 40 minutes from my home. I went through 6 years of chemo therapy and eventually found remission. I see a specialist every 3 months to keep an eye on my disease. In the past 3 years, I've been in treatment again, returning to chemotherapy and steroids to keep me from losing kidney function. It has been a difficult journey, but I am lucky because of some great doctors.
I am now 68 years old, and I work very hard at advocacy in my disease field. I don't want others to die from this disease because they could not be diagnosed."
#RareDiseaseWeek #MicroscopicPolyangiitis #ANCAVasculitis #WeLoveRare #RareNotDifferent #PatientWorthy
We are excited to share that Patient Worthy is headed to Washington, DC for Rare Disease Week! 🦓 💙
During Rare Disease Week, advocates from across the country gather to amplify the voices of the rare community, share powerful stories, and push for policies that improve access, research, and care. We're honored to stand alongside patients, caregivers, and other organizations making a difference.
If you'll be in DC next week, let us know in the comments! Let's keep raising awareness and driving change, together!
#RareDiseaseWeek2026 #RareDC2026 #PatientWorthy
United Leukodystrophy FoundationReminder that scholarship applications for the 2026 United Leukodystrophy Foundation (ULF) Family Conference are due by this Friday, February 27th! Applicants will be notified during the week of March 9th. To apply, please read the Scholarship Guidelines, complete and submit the application here: ulf.org/family-conference-scholarships/ #Leukodystrophy #ULFConference
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