Sjogren's Syndrome
Ronnie’s Journey: How a Lifelong Series of Symptoms Led to a Sjogren’s Diagnosis
As far as I can remember, I have always had an intolerance of heat and cold. I can remember at a young age not wanting
An Itch That Was Taking Over My Thoughts: A Story About Primary Biliary Cholangitis
- February 25, 2026
Dear Cancer: We Are Stronger Than You
AstraZeneca Pursues FDA Approval for Injectable Lupus Treatment Following European Authorization
Gretchen’s Battle with Salmonella Osteomyelitis
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Fabry Disease
From Invisible Pain to Unwavering Spirit: My Fabry Disease Journey
For most of my life — starting when I was eight years old — I lived with a deep burning pain in my hands that
Ovarian Cancer
Breaking New Ground: Immunotherapy Now Available for Recurrent Ovarian Cancer Patients
When ovarian cancer returns and becomes resistant to platinum-based treatments, patients face one of oncology’s most difficult scenarios. As reported by Drugs.com, a new therapeutic
Mosaic Variegated Aneuploidy syndrome 3
Navigating Survivorship and Self-Advocacy After Childhood Cancer: Maria’s Journey with Mosaic Variegated Aneuploidy Syndrome 3 (MVA 3)
My story began before I was old enough to understand it. Childhood cancer shaped my body and my life long before I had words for
Ovarian Cancer
A New Treatment to Fight Ovarian Cancer has been Developed by UCLA Scientists
CAR-NKT cell therapy (NKT) is capable of precision targeting when equipped with CAR. The UCLA study’s co-senior author, Dr. Lili Yang stated that personalized immunotherapies
Myositis
Cabaletta Bio Advances Rare Disease Treatment with Pioneering Myositis Trial and Innovative Manufacturing Breakthrough
Cabaletta Bio has announced significant progress in its development of chimeric autoantibody receptor T cell (CAAR-T) therapies, marking a major milestone with the initiation of
Rare Disease
After the Diagnosis: Self-Harming Thoughts and So Many Questions
After being diagnosed with a rare disease or any other chronic health condition (in my case I have dystonia) and learning it is not a
Graves' Disease
Thriving Is a Choice: How Community, Boundaries, and Mindfulness Saved My Life & Thyroid
Editor’s Note: Patient Worthy is proud to share part 10 of 10 of Elena Genik’s blog, detailing her journey with Graves disease and thyroid eye
ornithine transcarbamylase deficiency
Pruitt’s Legacy: Raising Awareness of OTC Deficiency and Genetic Testing
Rare Disease Month is deeply personal to our family. Before Pruitt, our youngest son, we had never heard of Ornithine Transcarbamylase (OTC) Deficiency, nor did
Stiff Person Syndrome
Celine Dion’s Stiff Person Syndrome Treatment and Progress
Ever since Celine Dion, the popular superstar, announced the cancellation of her 2023 tour due to a diagnosis of Stiff Person Syndrome, or SPS, the
Rare Disease
FDA Advances Rivoceranib-Camrelizumab Combination: A Promising Breakthrough for First-Line Unresectable Hepatocellular Carcinoma Treatment
Elevar Therapeutics has achieved a significant regulatory milestone with the US Food and Drug Administration’s acceptance of a resubmitted New Drug Application for rivoceranib in
Rare Disease
Nutcracker Syndrome, May-Thurner Syndrome, and More: Tytina’s Journey to a Proper Diagnosis
Sharing My Stripes 🦓 This Rare Disease Month, I’m honored to share my journey living as someone with a rare disease and what Rare Disease
Graves' Disease
Why Graves and Thyroid Eye Disease Are Immune System Disorders, Not Thyroid Disorders
Editor’s Note: Patient Worthy is honored to share part 9 of 10 of Elena Genik’s blog documenting her experiences with Graves disease and thyroid eye
Melanoma
FDA Listens: When Regulatory Bodies Recognize Humanity in the Fight Against Advanced Melanoma
When patients and families face a life-or-death disease, bureaucracy often feels like the enemy. Yet in early February 2026, the FDA delivered something rare: a
Acromegaly
Tessa’s Journey with Acromegaly
Hi! My name is Tessa, and I have a rare disease called Acromegaly. My journey started in 2015 when we discovered I had a 9x10x12
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
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
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Meet Patricia! For Rare Disease Month, Patricia shares her story in hopes of inspiring others. She is a liver transplant survivor, advocate, and voice for resilience. After being diagnosed with Primary Biliary Cholangitis at a young age, her health journey ultimately led to a life-saving liver transplant in 2018. Through sharing her story, Trish hopes to encourage others facing chronic illness to trust their instincts, stay informed, and never lose hope. Today, she uses her experience to uplift and empower others, raising awareness about organ donation, life after transplant, and the strength found in perseverance.
Read Patricia's full story here: bit.ly/4tGviOp
#rarediseasemonth #raredisease #rarebutnotalone #WeCareAboutRare #SHAREYOURSTRIPES #shareyourstory #PatientWorthy #PrimaryBiliaryCholangitis #PBC
Raynaud's disease affects blood flow to the fingers, toes, ears, and nose - often triggered by cold temperatures or stress. During an episode, skin may turn white, blue, then red, and can feel numb, tingly, or painful. 👐
It's more than "just cold hands." For many, Raynaud's impacts daily life in ways others may not see. Let's use this month to educate, advocate, and show understanding for those living with Raynaud's. #RaynaudsAwareness #StayWarm #PatientWorthy
Kathleen and Noah have been courageously navigating life with PPA2 deficiency. Patient Worthy is honored to share their story during Rare Disease Month and shine a light on their journey! Heart of PPA2
"Hi, I’m Kathleen and I’m Noah. Noah was diagnosed with a rare mitochondrial disease called PPA2 deficiency after experiencing four cardiac arrests before the age of two. Life with a rare disease was not something we ever imagined for our family, and yet here we are. Living with a rare disease like PPA2 means learning a diagnosis most doctors have never heard of, becoming an expert out of necessity, and carrying a constant awareness that life can change in an instant.
One of the most important things I want people to understand about PPA2 deficiency is that it is devastating and under-researched. It primarily affects the heart and energy production at a cellular level, and too often the first symptom is sudden cardiac arrest. There is no cure. There are no approved treatments. Families are left navigating survival with limited guidance, which is why research, awareness, and funding are not optional—they are urgent.
My hope for the future is for funded research, earlier diagnoses, and lifesaving treatments. I hope for a world where rare disease families are met with knowledge instead of confusion, and where children diagnosed with PPA2 deficiency are given a future instead of a prognosis.
Rare Disease Month is deeply personal to me. It’s a chance to speak up for a community that is too often overlooked, to put faces and names to diagnoses that are frequently dismissed because of their rarity. Participation matters because awareness leads to research, research leads to treatment, and treatment saves lives. For us, Rare Disease Month isn’t just about recognition…it’s about survival."
#RareDiseaseMonth #RareDisease #RareButNotAlone #WeCareAboutRare #ShareYourStripes #ShareYourStory #PatientWorthy #PPA2
