Cystic Fibrosis
Nick and Rene’s Caregiver Story for Their Daughter with Cystic Fibrosis
I’ve learned something brutal over the last few weeks: Most people cannot tolerate the reality of a child nearly dying. So they dilute it. They
Two-Decade-Old Cancer Vaccine Study Reveals Clues to Long-Term Survival
- February 3, 2026
When Healing Goes Wrong: How I Accidentally Triggered My Graves Disease
Why Most Cancer Patients Don’t Benefit from Modern Immunotherapy, And Why Your Heart Medication Might Change That
Moderna Partners With Recordati to Advance mRNA Therapy for Propionic Acidemia
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THE LATEST FROM PATIENT WORTHY
HIV/AIDS
From HIV Treatment to Rare Genetic Disease: Nucleoside Therapy Enters a New Era
As reported on Forbes, a class of drugs first introduced to combat HIV is now demonstrating unexpected promise for patients with rare genetic and mitochondrial
Psoriasis
Once-Daily Zasocitinib Delivers Clear Skin for Psoriasis Patients: Takeda’s Landmark Phase 3 Victory
Takeda Pharmaceutical Company has announced breakthrough results from Phase 3 trials of zasocitinib (TAK-279), an innovative oral treatment for moderate-to-severe plaque psoriasis that offers patients
Rare Disease
Biohaven’s BHV‑7000 Falls Short in Midstage Depression Trial, Refocusing Pipeline Strategy
As reported on FierceBioTech, Biohaven closed out the holiday period with disappointing news: its investigational Kv7.2/7.3 potassium‑channel modulator, BHV‑7000, did not demonstrate efficacy in a
Non-Hodgkin's Lymphoma
“The Aftermath: Nobody Told Me This Part Would Hurt Too: – Megan’s Fight Against Non-Hodgkin’s Lymphoma
Editor’s Note: Patient Worthy is proud to share this story from our friends at Elephants & Tea. To see the story in its original format,
Insomnia
Breaking New Ground: Daridorexant Offers Hope for Menopausal Insomnia
Menopause-related insomnia affects approximately one in four women during the menopausal transition, yet it remains significantly underdiagnosed and undertreated. A new analysis published in Maturitas
Hunter Syndrome
UNC Researchers Report Promising Advances Toward First Brain‑Targeting Enzyme Therapy for Hunter Syndrome
As reported by NewsWise, new clinical study led by investigators at the University of North Carolina at Chapel Hill is offering rare hope for families
Alzheimer's disease
Fast-Track FDA Review Accelerates Path to First Treatment for Alzheimer’s-Related Agitation
Axsome Therapeutics has reached a significant regulatory milestone with the US Food and Drug Administration’s acceptance of a supplemental New Drug Application (NDA) for AXS-05
Rare Disease
FDA Approves First At-Home Brain Stimulation Device for Depression
As reported by Forbes, the U.S. Food and Drug Administration has granted approval to Flow Neuroscience for the first prescription brain stimulation device designed for
Prader-Willi Syndrome
Breakthrough Treatment for Prader-Willi Syndrome Validated in Peer-Reviewed Journal
Soleno Therapeutics has achieved a significant milestone in the treatment of a rare genetic disorder, announcing the publication of pivotal clinical study results for VYKAT™
Achondroplasia
Our Achondroplasia Journey: What I Wish I’d Known When My Daughter Was Diagnosed
Editor’s Note: Patient Worthy is proud to share this caregiver story submitted to us by Missy Williams. Nine years later, I can still picture the
Bladder cancer
Bladder Cancer Treatment Landscape Shifts: ImmunityBio’s Long-Term Data Challenges Surgical Intervention Rates
ImmunityBio’s latest clinical findings reveal a significant development in the ongoing battle against non-muscle-invasive bladder cancer, with three-year follow-up data suggesting that combining Anktiva with
Lymphoma
A Conversation About Survivorship
Editor’s Note: Patient Worthy is proud to share this article, originally written by Katie Tat, from our friends at Elephants & Tea. To see the
Rare Disease
Advanced Brain Scans, and Chinese Medicine, Show Promise for Personalized Depression Care
As reported on Science Daily, a recent clinical study suggests that a traditional Chinese herbal remedy may offer a unique advantage in treating major depressive
Psoriasis
FibroBiologics Advances Innovative Cell Therapy: FDA Review of CYPS317 for Psoriasis Treatment Marks Major Clinical Milestone
FibroBiologics Inc, a clinical-stage biotechnology company, has announced a significant breakthrough in regenerative medicine by submitting a Phase 1/2 Investigational New Drug (IND) application to
Rare Disease
It Was All a Dream
Editor’s Note: Patient Worthy is honored to share Michelle Patidar’s patient story, provided to us by our friends at Elephants & Tea. To see the
Lupus
Geri’s Complicated Journey with Lupus, Sjogren’s and Non-Hodgkins Lymphoma
My chronic health journey is super complicated. When I first started experiencing my symptoms I wouldn’t tell anyone what I was feeling. I would wake
Rare Disease
From Cancer Treatment to Childhood Health: How Foresee’s Injectable Drug Addresses a Rare Pediatric Challenge
When pharmaceutical companies develop medications for serious diseases like prostate cancer, few anticipate how those treatments might transform care for entirely different patient populations. Yet,
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Education is the first step toward building a more inclusive world—replacing stares with support and curiosity with compassion.
Understanding the physical and genetic differences in Apert Syndrome helps ensure that every individual has the support they need to reach their full potential. Let’s keep learning together! 💙
#RareDiseaseOfTheWeek #ApertSyndrome #RareDiseaseAwareness #CraniofacialAcceptance #InclusionMatters #GeneticHealth
CELEBRATE! The BEST news! Thank you to all who helped make this possible EveryLife Foundation for Rare Diseases‼️Breaking News: Rare Pediatric Disease PRV Program Reauthorized by Congress!
After a two-year campaign to reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program, the rare disease community’s relentless advocacy has paid off.
Congress has passed the Labor, HHS, and Related Agencies Appropriations bill, effectively reauthorizing the PRV Program for five years while also funding a number of other critical healthcare agencies.
We applaud the reauthorization of the PRV Program and renewed investments in critical health research and public health programs.
Thank you to the congressional champions who have partnered with our rare disease community to secure these advances. While significant work remains to enable all those living with rare diseases to thrive, today’s progress will accelerate innovation, expand access to life-changing therapies, and offer renewed hope to children and families whose futures once seemed beyond reach.
To learn more about the full healthcare package, please visit our website: everylifefoundation.org/congress-passes-five-year-reauthorization-of-rare-pediatric-disease-prv-p...
Obesity is a medical condition affecting over 100 million Americans—yet it's often misunderstood. Swipe to understand what obesity actually is, how it differs from being overweight, and why the conversation matters. #obesity #PatientWorthy
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