Nipah Virus
Who is at Risk of Getting the Deadly Nipah Virus?
It took two new cases of the deadly Nipah virus to remind people in West Bengal, India of the 1998 outbreak that killed over one
Ronnie’s Journey: How a Lifelong Series of Symptoms Led to a Sjogren’s Diagnosis
From Invisible Pain to Unwavering Spirit: My Fabry Disease Journey
Breaking New Ground: Immunotherapy Now Available for Recurrent Ovarian Cancer Patients
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THE LATEST FROM PATIENT WORTHY
Narcolepsy
FDA Grants Priority Review to Takeda’s Oveporexton: A Potential Game-Changer for Narcolepsy Type 1 Treatment
Takeda Pharmaceutical has reached a significant milestone in the development of oveporexton (TAK-861), with the US Food and Drug Administration officially accepting and granting Priority
Metabolic dysfunction-associated steatohepatitis
CHMP Backs Novo Nordisk’s Semaglutide for MASH, Opening Door to EU Approval
As reported on MedicalXpress, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for several therapies this
Graves' Disease
Rock Bottom in Mexico: The Thyroid Eye Disease Flare That Forced Me to Rebuild My Health
Editor’s Note: Patient Worthy is proud to share part 7 of 10 of Elena Genik’s blog series, detailing her experiences with Graves disease and thyroid
Duchenne Muscular Dystrophy
Aucta Pharmaceuticals Launches PYQUVI, Expands Branded‑Generic Portfolio in Rare Disease Care
As reported on the Manila Times, Aucta Pharmaceuticals has officially introduced PYQUVI™ (deflazacort) oral suspension 22.75 mg/mL, marking the company’s entry into the U.S. commercial
Duchenne Muscular Dystrophy
CureDuchenne’s 2026 Schedule
CureDuchenne is hosting several free events in 2026 to support families living with Duchenne and Becker muscular dystrophy. These events, called CureDuchenne CARES, will take
Eye Disease
Faricimab Delivers Durable, Real-World Benefits for Retinal Vein Occlusion
Twelve‑month, real‑world data from a large U.S. registry suggest that faricimab provides effective, durable, and safe treatment for retinal vein occlusion (RVO), reinforcing findings from
ADHD
FDA Prioritizes Review of Otsuka’s Centanafadine for ADHD Treatment
The U.S. Food and Drug Administration has agreed to review Otsuka Pharmaceutical’s application for centanafadine, a new extended‑release therapy under investigation for attention‑deficit hyperactivity disorder
Graves' Disease
The Emotional Toll of Thyroid Eye Disease: What Patients Are Not Told
Editor’s Note: Patient Worthy is honored to share part 6 of 10 of Elena Genik’s series of blog posts detailing her journey with Graves disease
Obesity
Dual Targeting Psoriatic Arthritis and Obesity: Taltz–Zepbound Combo Sets New Benchmark in TOGETHER-PsA Trial
A first-of-its-kind Phase 3b study has shown that combining Eli Lilly’s Taltz (ixekizumab) with Zepbound (tirzepatide) delivers superior benefits for adults living with both active
Follicular lymphoma
Triplet Therapy Strategies Signal a Shift in Relapsed Follicular Lymphoma Care
As reported by Bioengineer, new insights published by Nastoupil L.J. in Nature Reviews Clinical Oncology (2026) highlight an important evolution in the management of relapsed
Myelofibrosis
How Anemia Could Signal a Rare Blood Cancer
Editor’s Note: Patient Worthy is honored to share this article from our friends at Heal Canada, written by Karen Hawthorne. Anemia is one of the
Eye Disease
Optic Nerve Stem Cells Redraw the Map of Retinal Vascular Repair
A new study in Nature Communications and reported by Scienmag.com overturns a central assumption in eye biology by showing that key stem cells for the
Triple-negative breast cancer
Datroway Receives U.S. Priority Review for First‑Line Treatment of Metastatic Triple‑Negative Breast Cancer
In a recent statement by AstraZeneca, AstraZeneca and Daiichi Sankyo’s antibody‑drug conjugate Datroway (datopotamab deruxtecan) has been granted Priority Review by the U.S. Food and
Graves' Disease
When “Normal Labs” Are Not Normal: What Thyroid Patients Need to Know
Editor’s Note: Patient Worthy is pleased to share part 5 of 10 in an ongoing series of blog posts, provided to us by Elena Genik.
Skin Disease
Swiss Biotechs Gain Momentum with FDA Fast Track Option for Palmoplantar Pustulosis
A trio of Swiss biotechnology companies is advancing diverse therapeutic pipelines, with recent developments spanning inflammatory skin disease, immune‑mediated hair loss, and hard‑to‑treat cancers. Together,
Propionic Acidemia
Moderna and Recordati Partner on mRNA‑3927 for Propionic Acidemia: Clinical Development Advances Toward Pivotal Readout
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
Cancer
Nanoparticles Targeting Tumors Eliminates Most Chemotherapy Side Effects
Scientists find polymer-coated nanoparticles together with therapeutic drugs offer promise for cancer treatment, including treatment for ovarian cancer. According to Science Daily, these nanoparticles may
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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
