Rare Disease
In Rare Disease Care, Coordination Should Be Common
Editor’s Note: Patient Worthy is honored to share this submission from Katie DiLorenzo, SVP, Patient and Pharmacy Services, PANTHERx® Rare. If you’re living with a
Connor’s Leukodystrophy Story
The Quiet Revolution: When Hospitals Stop Being Your Second Home
FDA Expands Approval of Bizengri for Ultra-Rare Cholangiocarcinoma Subset
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Epidermolysis Bullosa
Solving Cell Therapy’s Dirty Secret: How One Partnership Could Unlock Consistent Treatments
Mesenchymal stromal cells (MSCs) have tantalized the medical world for years. These versatile immune-modulating cells offer genuine hope for diseases ranging from epidermolysis bullosa, a
Cancer
Nutrition and Cancer 101 – The Basics
Patient Worthy is honored to reprint this selection from The Cancer Dietitian, by Julie Lanford MPH, RD, CSO, LDN. It’s important to know that no single
Crohn's disease
Fighting for Himself and Others: Wojciech’s Battle Against Crohn’s Disease and More
I am sharing my journey to give a voice to patients who suffer from extreme poly-pathology and are often left behind by the medical system.
Colorectal cancer
Pre-Surgery Immunotherapy Rewrites Colorectal Cancer Survival: Zero Recurrence at Three Years
For decades, colorectal cancer treatment has followed the same playbook: surgeons remove the tumor, then patients endure months of chemotherapy hoping to catch any remaining
Parkinson's Disease
AskBio Advances Parkinson’s Gene Therapy Program With Commercial-Scale Manufacturing
AskBio Inc., a gene therapy company operating independently under Bayer AG, has taken a key step forward in the development of its investigational Parkinson’s disease
Lupus
Dismissal, Derision, and Misdiagnosis: Olga’s Lupus Story
Editor’s Note: This story was originally published on Linked-In and is shared with permission by Olga Lucia Torres. In college, a clinician told me I
Lupus
Breaking Ground: New Targeted Lupus Treatment Enters Critical Phase 3 Testing
Enpatoran offers hope for millions suffering from lupus skin manifestations Pharmaceutical company Merck KGaA has announced a significant milestone in lupus treatment research. As reported
Obesity
Vanda Initiates Thetis Trial to Address GLP‑1–Related Vomiting
As reported on PharmaBiz, Vanda Pharmaceuticals has launched a new clinical trial, known as Thetis, to evaluate whether its neurokinin‑1 (NK‑1) receptor antagonist Nereus (tradipitant)
Pancreatic Cancer
After Losing His Mother to Pancreatic Neuroendocrine Tumors, Michael Fitzpatrick Is Now a 26-Year Survivor of the Same Condition
Editor’s Note: Patient Worthy is honored to present this article, shared with us by our friends at the Seena Magowitz Foundation. When doctors diagnosed Michael
Epidermolysis Bullosa
Ireland Embraces Hope: Revolutionary EB Gel Treatment Now Available Through HSE
A major breakthrough for the 300 Irish patients living with butterfly skin. For the first time in Ireland, patients battling epidermolysis bullosa, a rare and
Rare Disease
Two Mothers. One Trial. Two Different Outcomes: A Mother’s Day Reflection from Clinical Research
Editor’s Note: Patient Worthy is honored to share this story submitted to us by Regina Portnoy, a clinical researcher with over 20 years of experience.
Osteosarcoma
The Timeless Passages of My Mind and Soul
Editor’s Note: Patient Worthy is honored to share this patient story from our friends at Elephants & Tea. To see the story in its original
Rare Disease
Topical Curcumin Gel Shows Promise for Knee Osteoarthritis Pain in Early Clinical Trial
As shared on drugs.com, Vascarta Inc. has reported encouraging results from a Phase 1b clinical study evaluating its investigational topical/transdermal curcumin gel, VAS-101, for the
Generalized arterial calcification of infancy-1 (GACI1)
Sir’Savion’s Story
Editor’s Note: Patient Worthy is honored to share this article from our friends at the Brighter Hope Foundation. To see the article in its original
Ovarian Cancer
Labcorp Brings Breakthrough Diagnostic to Identify Platinum-Resistant Ovarian Cancer Patients
Labcorp announced the nationwide availability of Agilent Technologies’ PD-L1 IHC 22C3 pharmDx, the only FDA-approved companion diagnostic designed to identify patients with platinum-resistant ovarian cancer
Pancreatic Cancer
Daraxonrasib Delivers Landmark Survival Gains in Previously Treated Metastatic Pancreatic Cancer
As reported on drugs.com, Revolution Medicines has reported practice-changing efficacy results from a pivotal Phase 3 trial evaluating daraxonrasib in patients with metastatic pancreatic ductal
Primary Biliary Cholangitis
The PBCers Conference
This, year the PCBers will meet in Indianapolis, Indiana. They will be celebrating 30 years as a community, as well as celebrating a much brighter
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A diagnosis can change everything — but for individuals living with Epidermolysis Bullosa (EB), getting answers is often the first step toward proper care, support, and improved quality of life. Because EB affects the skin on a genetic level, specialized testing helps identify the exact type, guide treatment decisions, and monitor complications early. Awareness and education matter, especially for rare conditions that are often misunderstood. Every diagnosis tells a story, and every story deserves understanding, advocacy, and hope. 💙
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