Charcot-Marie-Tooth disease
Can the Cost of Having a Rare Disease Be Calculated?
Editor’s Note: Patient Worthy is pleased to share this article, submitted to us by George Simpson, who volunteers for the CMT Research Foundation. There are
Nutcracker Syndrome, May-Thurner Syndrome, and More: Tytina’s Journey to a Proper Diagnosis
- February 19, 2026
Why Graves and Thyroid Eye Disease Are Immune System Disorders, Not Thyroid Disorders
FDA Listens: When Regulatory Bodies Recognize Humanity in the Fight Against Advanced Melanoma
Tessa’s Journey with Acromegaly
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Primary Biliary Cholangitis
IQIRVO: A Revolutionary PPAR Agonist Transforms Second-Line Treatment for Primary Biliary Cholangitis
Primary biliary cholangitis (PBC) represents a chronic, progressive autoimmune liver disease characterized by the destruction of small bile ducts, leading to cholestasis and potentially fatal
Myelofibrosis
We Will Never Ring the Bell – My Myelofibrosis Story
Editor’s Note: The above photograph is credited to Patient Voice. “Ringing the bell” is an enduring image associated with cancer survivorship. It represents victory, celebration
Graves' Disease
What Functional Medicine and Holistic Care Taught Me About Healing Graves and TED
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 and thyroid eye
ADHD
Beyond the Diagnosis: Otsuka’s “All of ADHD” Campaign Reframes Healthcare Provider Perspectives on a Complex Condition
Otsuka has launched an ambitious awareness initiative designed to fundamentally shift how healthcare professionals understand and approach attention-deficit/hyperactivity disorder (ADHD). As reported by FiercePharma.com, the
Activated PI3K delta syndrome
When Two Rare Diseases Collide: What My Son Taught Me About Immune System Complexity
When my son was eight months old, he had already been hospitalized more times than I can count for infections that seemed far too severe
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
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
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During Rare Disease Month, Patient Worthy is honored to share a story from Mary, who has been diagnosed with Granulomatosis with polyangiitis (GPA), formerly known as Wegener's disease.
"My name is Mary, and I am 71 years young. I am a wife, mother, and grandma. After working as an LVN/Nurse for 43 years, I retired. I love family time, cooking, gardening, and stained glass. My autoimmune disease started probably long before being diagnosed on April 4, 2022. I was in the hospital again, this time for kidney failure, and my kidney biopsy came back positive for Wegener's disease (GPA)–vasculitis. It has involved my kidneys, heart, lungs, eyes, and hearing. After two years of treatments and Peritoneal Dialysis (PD), I was blessed with healing. I healed enough to be able to stop dialysis. Many symptoms of the disease continue, so I continue to do oral treatments for immune therapy as recommended for me. Empower yourselves. Ask questions, be strong, and fight for your cure in your autoimmune disease."
#RareDiseaseMonth #RareDisease #RareButNotAlone #WeCareAboutRare #ShareYourStripes #ShareYourStory #PatientWorthy #Wegenersdisease #vasculitis #GPA
During Rare Disease Month, Patient Worthy is honored to share Gabby's story. Gabby's Wonderful World
Gabby is a 33-year-old school psychologist from Wading River, NY whose life is rooted in purpose, perseverance, and heart. She loves summer days at the beach and finds her greatest joy in her mini goldendoodle, Bella Grace, who is truly her whole world. Diagnosed with Neutral Lipid Storage Disease, an ultra-rare genetic disease, at 16 after a decade of misdiagnosis, Gabby began developing symptoms at 20—first in her arms, then her legs—leading to significant physical weakness that impacts her daily life. Walking and moving have become extremely difficult. Still, she refuses to be defined by her diagnosis. Every day, Gabby rises with intention, powering through long workdays, intensive therapies including PT, OT, and strength training, and leading Gabby’s Wonderful World Nonprofit Foundation. Her strength is not measured by what her body can do, but by the resilience, compassion, and purpose she brings into the world.
What Rare Disease Month Means to Gabby:
"Rare Disease Month is about visibility, advocacy, and hope. It is a reminder that even in the face of uncertainty and challenge, our lives carry immense value—and when we share our stories, we turn struggle into strength and isolation into community."
#RareDiseaseMonth #RareDisease #RareButNotAlone #WeCareAboutRare #ShareYourStripes #ShareYourStory #PatientWorthy #NeutralLipidStorageDisease
Kabuki Syndrome FoundationOn February 26th, the Kabuki Syndrome Foundation will host a scientific symposium for researchers around the world studying Kabuki syndrome - it is the only scientific meeting focused exclusively on Kabuki syndrome research!
This is one example of how KSF fosters collaboration in the scientific community to accelerate impactful research. While the event is intended for researchers, we’ll share a family-friendly summary afterward so you can stay up to date on the latest discoveries.
Sign up for our newsletter to receive key updates like our scientific symposium summary: bit.ly/KSFNewsRegister
