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,
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
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
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
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
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
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
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
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
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
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
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
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
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
Moderna and Merck have announced promising five-year data for a groundbreaking personalized cancer treatment that combines mRNA technology with immunotherapy to prevent melanoma from returning
As reported on Science Daily, a breast cancer vaccine tested more than 20 years ago is drawing renewed scientific interest after researchers discovered that every
Editor’s Note: Patient Worthy is honored to share part 2 in an ongoing 10-part series, written and shared with us by Elena Genik. Before the
Cancer immunotherapy represents one of modern medicine’s greatest achievements, yet it’s also one of its most frustrating paradoxes: a treatment that works miracles for some
For 10 years, ages five through 15, Erica watched her face change and become asymmetrical as a tumor like formation protruded from her face just under her left eye. It caused her eye to close up and put pressure on her right eye, which then bulged. In addition to the physical changes, she endured debilitating migraines, extreme nausea, random vision loss, breathing issues, slurred speech and weakness of her limbs. Despite visits to doctors, hospitals and specialists over that ten-year span, no one was able to tell her family what was happening. Finally, she was diagnosed with the rare genetic disorder Arteriovenous Malformation, aka AVM, and surgery was completed a few weeks later.
The majority of AVMs are usually located in the brain, and some people don’t know until there’s a rupture/hemorrhage. In Erica’s case, her family knew something was wrong because they could see the physical changes in her face, so they immediately began seeking medical advice. In 2019, a year after her surgery, Erica contacted The Aneurysm and AVM Foundation and founded their Youth Ambassador Program, which now has a network of young survivors and family members nationwide. Her main drive behind this program was to find other young survivors and their families, so they would know they’re not alone. With less than 1% of the population suffering from this disorder, it is almost next to impossible to meet people who have been through it or understand what you’ve been through, so it’s a very lonely journey.
Now, eight years after her life-changing surgery, Erica‘s life has truly changed. She’s much healthier, able to do a lot of activities that she could not participate in prior to the surgery, and she has spent the past seven years advocating for the AVM community. Through TV/newspaper/magazine/online interviews, where she shares her story and spreads AVM awareness, Erica’s goal is to help struggling families identify with the symptoms she discusses and share the info with their doctors. If she had seen just one person sharing their AVM story on TV or in a magazine, she could’ve quickly identified with their symptoms and asked her doctor if maybe this was the problem. As we acknowledge Rare Disease Month and Rare Disease Day worldwide, always remember that you’re not alone. In the AVM community our tag line is, “From survivors to caregivers, researchers to advocates, we are #UnitedAndRare — because it’s only RARE until it happens to you!”
Share your rare disease story with us here: bit.ly/4dV7gru
#RareDiseaseWeek2026 #ArteriovenousMalformation #PatientWorthy
Ectodermal Dysplasia Awareness Month is a chance to learn about the genetic conditions that affect hair, teeth, skin, nails, and sweat glands. People living with ectodermal dysplasia often navigate challenges like temperature regulation and dental or skin differences. By spreading awareness, we help build understanding, support, and early recognition for those affected. 💛 #ectodermaldysplasiaawareness #rarediseaseawareness
During Rare Disease Month, Patient Worthy is honored to share Krystle's journey!
Krystle is a 33‑year‑old mother, storyteller and advocate living with myositis. This rare autoimmune disease causes her immune system to attack her muscles, which leads to inflammation, weakness and chronic fatigue. At this stage of life, she navigates motherhood in ways she never expected. Her two daughters support her emotionally and sometimes step in as caregivers when her body needs rest. Tasks that once felt automatic now take planning, patience and creativity. Mobility looks different. Her energy is limited, so she’s learned to protect it and focus on what truly matters. Through motherhood, work and community building, she shows that strength is more than physical. It’s resilience, intention and heart.
For Krystle, Rare Disease Month is about visibility and truth. Rare does not mean insignificant, and muscle weakness does not mean weak. By sharing her story, she hopes to raise awareness, challenge assumptions about invisible illness and remind others living with rare conditions that their experiences matter. Advocacy, for her, isn’t just about being heard. It’s about making sure no one feels overlooked or alone.
#RareDiseaseMonth #RareDisease #RareButNotAlone #WeCareAboutRare #ShareYourStripes #ShareYourStory #PatientWorthy #MyositisAwareness #AutoimmuneWarrior
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