Rare Disease
Advances in Stroke Recovery
If you or a loved one has ever survived a stroke, you know that the hardest part of the journey often begins after you leave
Teen Caregiver Finds New Purpose: Lilly’s Aplastic Anemia Story
Efgartigimod Shows Promise in Immune Checkpoint Inhibitor-Related Myasthenia Gravis–Myositis Overlap Syndrome
Pembrolizumab Outperforms Standard Chemotherapy in First-Line dMMR Endometrial Cancer Study
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THE LATEST FROM PATIENT WORTHY
Rare Disease
Leadership Is More Than a Title
Editor’s Note: This article was originally written and submitted to us by Regina Portnoy. When people think about leadership in clinical research, they often think
Chronic Kidney Disease
Some Dialysis Humor, Part 3
Editor’s Note: This is part 2 of a 3-part caregiver story, submitted to us by Joan Foster, who was a caregiver for her husband Charles’
Rare Disease
Repairing the Wiring: How a New Stem Cell Therapy Aims to Defeat Chronic Spinal Cord Injury
As reported on Inside Precision Medicine, for years, a chronic spinal cord injury (SCI) has felt like a permanent biological roadblock. When the spinal cord
Major Depressive Disorder
Joan’s Journey with MDD
Thoughts occur in the mind, when the electrical circuits in the brain through neurons and synapses and chemicals all function together. Processes of the mind
Melanoma
Huyabio’s HBI-8000 and Opdivo Combination Shows Significant Benefit in Phase 3 Melanoma Study
As reported on Fierce Pharma, Huyabio has reported encouraging Phase 3 results for its investigational oral HDAC inhibitor, HBI-8000, when used alongside Bristol Myers Squibb’s
Megan’s Myasthenia Gravis Story: From Uncertainty to Advocacy
I was diagnosed with myasthenia gravis, or MG, in 2021 when I was in my early 20s. My journey began at a friend’s birthday party
Chronic Kidney Disease
Some Dialysis Humor, Part 2
Editor’s Note: This is part 2 of a 3-part caregiver story, submitted to us by Joan Foster, who was a caregiver for her husband Charles’
Major Depressive Disorder
AtaiBeckley Completes Patient Dosing in Phase 2b Trial of VLS-01 for Treatment-Resistant Depression
As reported on PharmaBiz, AtaiBeckley has announced a key milestone in the development of its investigational depression therapy, VLS-01, with the final participant now dosed
Generalized arterial calcification of infancy-1 (GACI1)
Natalie’s GACI Story
Editor’s Note: Patient Worthy is honored to share this story, originally written by Anne and Jerry van Wyk, and shared with us by our friends
Rare Disease
“Two Rare Diagnoses, One Common Lesson: Start with Genetic Testing”
Every day, parents just like me sit in doctors’ offices searching for answers. They know something isn’t quite right. Maybe their child isn’t meeting milestones.
Mesothelioma
New Experimental Drug Offers Hope for Mesothelioma Patients
As reported on Vermont Biz, researchers at the University of Vermont (UVM) and biotechnology company RS Oncology have reported encouraging results from an early-stage clinical
Alzheimer's disease
2026 Alzheimer’s Update
For more than a century Alzheimer’s disease has been thought to be incurable. But a recent study published in the New England Journal of Medicine
Chronic Kidney Disease
Some Dialysis Humor, Part 1
Editor’s Note: This is part 1 of a 3-part caregiver story, submitted to us by Joan Foster, who was a caregiver for her husband Charles’
Hepatocellular Carcinoma
FDA Issues Complete Response Letter for Rivoceranib–Camrelizumab Combination in Advanced Liver Cancer
As reported by The Manila Times, the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to Elevar Therapeutics regarding its
Hemophilia A
ISTH 2026: Long-Term Phase 3 Data Support Denecimig as a Flexible Prophylactic Option for Hemophilia A
As covered by Fairfax Times, Novo Nordisk has reported encouraging long-term findings for its investigational hemophilia A therapy denecimig (Mim8), with interim results from the
Rare Disease
Heal Canada’s Summer 2026 Issue of E3 Advocacy Magazine
Our friends at Heal Canada have launched the newest edition of their magazine, E3 Advocacy! Their digital publication focuses on the importance of patient advocacy,
Rare Disease
FDA Grants Breakthrough Device Designation to Aurenar’s V-Link System for Preventing Cerebral Vasospasm After Brain Hemorrhage
As reported on MobiHealth News, the U.S. Food and Drug Administration (FDA) has awarded Breakthrough Device designation to Aurenar’s V-Link System, a noninvasive neuromodulation technology
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United Porphyrias AssociationWhat exactly is an enzyme, and why does it matter in porphyria?👀 Think of enzymes as tiny workers on a production line. Their job is to help your body make heme, an essential molecule your body needs every day.
In porphyria, one of those "workers" doesn't function properly ⚠️ When that happens, the production line slows down, and porphyrins or their precursors begin to build up. That buildup is what leads to the symptoms of porphyria.
Understanding how enzymes work helps explain why there are different types of porphyria—and why each one is caused by a problem in a different step of the heme-making process.
💬 What would you like #ProfessorPorphy to teach next? Leave your porphyria questions in the comments, and your question might be featured in a future class!📚
#education #porphyria #raredisease #enzymes
Living with a chronic illness often means navigating doctor appointments, medications, treatments, symptoms, insurance hurdles, and the unpredictability that comes with managing a long-term condition. It's not about being "high maintenance"—it's about doing what it takes to care for your health and well-being.
For millions of people living with chronic and rare diseases, the daily effort behind the scenes can be exhausting, invisible, and often misunderstood. A little compassion, patience, and understanding can go a long way.
#ChronicIllness #InvisibleIllness #RareDisease #ChronicDiseaseAwareness #PatientWorthy #ShareYourStory
