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
The Emotional Toll of Thyroid Eye Disease: What Patients Are Not Told
Dual Targeting Psoriatic Arthritis and Obesity: Taltz–Zepbound Combo Sets New Benchmark in TOGETHER-PsA Trial
Triplet Therapy Strategies Signal a Shift in Relapsed Follicular Lymphoma Care
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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
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February is Rare Disease Awareness Month, a time to recognize the more than 30 million people in the U.S. living with one of thousands of rare conditions and the unique challenges they face. Because rare diseases often impact emotional well‑being, finances, and daily life in ways that aren’t always visible, understanding how to support someone living with one is incredibly important. Many people experience long diagnostic journeys, misunderstood symptoms, and social isolation, which can make the world feel smaller and harder to navigate. When friends, family, and communities take the time to learn, listen, and show compassion, it helps rare disease patients feel seen, validated, and less alone in a journey that can often feel overwhelming. This month is a reminder to stand with the rare disease community and help create a world where no one feels invisible. 💙
#RareDiseaseMonth #RareDisease #RareButNotAlone #WeCareAboutRare #ShareYourStripes #ShareYourStory #PatientWorthy
Meet Samantha who is living life with Loeys Dietz Syndrome and Homocystinuria. This Rare Disease Month, her story helps remind us why representation, awareness, and community matter!
"Hi, my name is Samantha. I have Loeys Dietz Syndrome and Homocystinuria. It has been 10 years since I was diagnosed with both rare diseases at the same time. I was missed through the Newborn Screening for HCU, as back in 1992 when I was born where I am from did not have HCU on the NBS and I was missed for 23 years. While HCU has been around for a long time, LDS has only been around since 2006. My parents and my cardiologist always knew there was something going on, but we all could never pinpoint what it was. During my spring semester of my senior year in college, I felt something was off, and I had mentioned this to my mom and neurosurgeon, as I also have Chiari. My neurosurgeon did all his tests and could not find anything, and I was then sent to the genetics department and that is when I got blood drawn for a full genetic panel. I was officially diagnosed months later after fighting with insurance that this was medically necessary. About six months after my diagnosis, I was trying to find ways to get involved and meet others within the rare disease community as I have always done better when I am able to find a support group of friends. That is when I found the Everylife Foundation and joined their young adult program. In February of 2019, I started my rare disease advocacy journey by going to Rare Disease Week on Capitol Hill. Ever since then, I have advocated with my local and federal legislators on behalf of rare disease legislation. I advise those who are just starting in their rare journey to never give up the fight, whether it is for your own healthcare or helping others by advocating with your local and federal legislators. Rare Disease Month means awareness, awareness that my friends and I are seen, because we are not one, but many hoofbeats of mighty zebras!"
#RareDiseaseMonth #RareDisease #RareButNotAlone #WeCareAboutRare #ShareYourStripes #ShareYourStory #PatientWorthy #LoeysDietzSyndrome #Homocystinuria
We're honored to continue sharing Elena Genik's story about her experiences with Graves disease and Thyroid eye disease. Read part 6 of 10 here:
patientworthy.com/2026/02/12/the-emotional-toll-of-thyroid-eye-disease-what-patients-are-not-told/
