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
Dual Approach to Disease: Eli Lilly’s Zepbound-Taltz Combination Demonstrates Breakthrough Results in Psoriasis Treatment
- February 27, 2026
My Journey with Limb-Girdle Muscular Dystrophy (LGMDR1)
LISTEN TO OUR PODCAST
THE LATEST FROM PATIENT WORTHY
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
Eye Disease
Turning Heart Data Into an Early Warning System for Vision Loss
A UCLA Health study published on UCLAHealth.org suggests that numbers already used to gauge heart health may also offer an early warning for serious eye
Cancer
New Immunotherapy Strategy Targets Tumor ‘Bodyguards,’ Shows Powerful Results in Preclinical Cancer Models
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
Graves' Disease
Medical Gaslighting Is Real: The Appointment That Changed Everything
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
MPS I (Hurler Syndrome)
FDA Places Clinical Holds on REGENXBIO’s MPS Gene Therapy Programs After Tumor Detected in Trial Participant
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
Myelofibrosis
James’ Story: Navigating Life with Myelofibrosis
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
Obesity
Roche’s Obesity Drug Shows Promise Against Lilly and Novo Nordisk
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
Squamous-cell carcinoma
EMA Endorses First Immunotherapy Option for Advanced Anal Cancer
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
Graves' Disease
The Invisible Battle No One Warned Me About: Developing Thyroid Eye Disease
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
Thyroid eye disease
Artificial Intelligence Advances Eye Disease Diagnosis: Deep Learning Shows Promise in Distinguishing Thyroid Eye Disease from Orbital Myositis
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
Hunter Syndrome
FDA Places Clinical Hold on REGENXBIO’s MPS Gene Therapy Programs After Tumor Case Review
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
Pancreatic Cancer
An International Team of Researchers Identify a New Key Factor Influencing Pancreatic Cancer
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
FEATURED
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
During Rare Disease Month, Patient Worthy is honored to share Diana's journey.
Having a rare disease has been devastatingly life-altering. I went from being an aircraft mechanic, OTR driver, and most recently, in 2020, a caregiver when the disease manifested violently during COVID. For over five years and counting, I have been completely bedridden. My proudest roles as a wife, mother, daughter, sister, and Mamaw have all been affected because of my limitations. As someone who has always taken care of her family, I am no longer able to provide for them now.
Since discovering my condition, I've learned that this disease has manifested itself over a lifetime, and since COVID, it has become more aggressive and detrimental to my health with new conditions emerging in a matter of months. Not enough doctors know about rare diseases, but I was lucky enough to have found doctors that advocated for me. One in particular ran tests, talked to other doctors in various fields, and simply took the time to talk to me. Not everyone gets that luxury, and I am so thankful.
Having a rare disease has also taught me patience, understanding, and forgiveness. I did have these before, but not at the depth I do now. It has also given me time—time to spend with my husband, something I rarely had before. Time when my grandbabies come over to lie in bed and cuddle while watching a show. Something I would miss doing if I were still a workaholic.
I have learned that we are our own best advocates. Make sure your voice is heard. Take notes, keep a journal, and use a calendar so that you can document your symptoms, your feelings, and questions you think of at any given moment. Please ask your questions! This is about your health.
I have a rare disease. I still have dreams that one day I can get up and move without being in pain every moment. My hope is that more knowledge and resources are spread and made accessible to those fighting this battle with me.
#rarediseasemonth #raredisease #rarebutnotalone #WeCareAboutRare #SHAREYOURSTRIPES #shareyourstory #PatientWorthy
Idiopathic Intracranial Hypertension (IIH) is more than “just a headache.” It’s a rare neurological condition where pressure builds around the brain without a known cause — impacting vision, mobility, energy, and daily life. Many people with IIH fight silent battles with pain, uncertainty, and misunderstood symptoms every single day.
Meet Mckayla who is diagnosed with Idiopathic Intracranial Hypertension. Patient Worthy is honored to share her story during Rare Disease Month!
"Hello, my name is Mckayla Rees and I'm 18 years old. I love gaming and hanging out with people. I have idiopathic intracranial hypertension which is a build up of spinal fluid in your head. I've had a lot of symptoms like headaches but I've learned it gets better. My disease means trying even though it's hard."
#RareDiseaseMonth #RareDisease #RareButNotAlone #WeCareAboutRare #ShareYourStripes #ShareYourStory #PatientWorthy #IdiopathicIntracranialHypertension #IIH
Did you know myositis can be triggered by various factors including other autoimmune diseases, infections, or even injuries? Sometimes it occurs on its own, and other times it's linked to medication side effects. Having to navigate a complex web of symptoms can be a lot. By sharing your story, you can help others understand what it's like to live with this chronic condition and join a community of support. Click the link below and share with us today! bit.ly/3VuxFDV #invisibleillness #raredisease #PatientWorthy
