Heart disease
Choosing to Be Me: Redefining My Life with Rheumatic Heart Disease
My diagnosis of rheumatic heart disease came on suddenly. I remember being 12, sitting at the doctor’s office, and was told that I have a
Akebia Doses First Volunteers in Phase 1 Study of AKB-9090 for Surgery-Related AKI
- April 15, 2026
Triple Negative Breast Cancer: A Breakthrough Treatment
Combinatorial Immunotherapy Strategy: CatalYm Advances Visugromab in Advanced Liver Cancer
Litifilimab Shows Promise in Phase 2 Trial for Cutaneous Lupus
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Huntington's disease
Major Breakthrough: Discovery of a Pathway that Allows Brain Cells to Move Toxic Material in Huntington’s Disease
Researchers at Florida Atlantic University (FAU) have identified a cellular pathway that allows brain cells to share proteins and other toxic material. The study was
Narcolepsy
Lupin Receives FDA Approval for Pitolisant: A Novel Solution for Narcolepsy Management
Lupin, a globally recognized pharmaceutical company, has achieved a significant regulatory milestone with the receipt of tentative FDA approval for Pitolisant tablets in strengths of
Diabetes Type 2
Can Your Morning Coffee Really Help Prevent Type 2 Diabetes?
Editor’s Note: Patient Worthy is honored to present this article, shared with us by our friends at the Steatotic Liver Foundation (formerly the Fatty Liver
Huntington's disease
Heather and Nathan’s Journey with Huntington’s Disease
Fight. I don’t think that those who aren’t in the Huntington’s Disease community understand what the word FIGHT truly means to those of us inside
Ehlers-Danlos Syndrome
My Journey with Ehlers-Danlos Syndrome
My name is Amanda. I was diagnosed with a genetic connective tissue disease called Hypermobile Ehlers-Danlos Syndrome (h-EDS) about four years ago. However, the diagnosis
Amyloidosis
Faye’s Journey with Amyloidosis
My name is Faye. I’m blessed to be the wife of the love of my life, Brad, and the proud mom of two extraordinary boys
Obesity
Setmelanotide Shows Limited Efficacy in Heterozygous Obesity Gene Carriers
A major clinical trial has revealed disappointing results for setmelanotide, a promising obesity treatment, when used in patients carrying single genetic mutations associated with weight
Cancer
Illumina and Labcorp Deepen Collaboration to Broaden Access to Precision Oncology Testing
As reported on PharmaBiz, Illumina and Labcorp have announced an expanded strategic collaboration aimed at accelerating precision oncology by applying next-generation sequencing (NGS) technologies more
Ehlers-Danlos Syndrome
Opinion Piece: Insurance Abuse
Editor’s Note: The views, thoughts, and opinions expressed in this article belong solely to the author and do not necessarily reflect the position of Patient
non-small cell lung cancer
Eftilagimod Alfa Setback: Immutep Halts Landmark Lung Cancer Trial Following Futility Analysis
Immutep Limited has announced the discontinuation of TACTI-004, a pivotal Phase III clinical trial investigating eftilagimod alfa (efti) as a first-line treatment for advanced non-small
non-small cell lung cancer
Dizal’s Zegfrovy Delivers Phase 3 Breakthrough in EGFR Exon 20–Mutant NSCLC
As reported on FiercePharma, China-based biotech Dizal Pharmaceutical has reported a pivotal phase 3 success for its oral EGFR inhibitor Zegfrovy (sunvozertinib), marking a potential
Renal cancer
The Luck of the Irish: A Parable of Inheritance, Illness, and Hope
Editor’s Note: The following article was originally written by Kelly Curtin-Hallinan, and shared with us by our friends at Elephants & Tea. This past St.
RSV
FDA Approves RSV Vaccine for Younger Adults: What You Need to Know
Arexvy Now Available for Adults Aged 18–49 with Certain Health Conditions The U.S. Food and Drug Administration has granted approval to expand GSK’s Arexvy respiratory
Rare Disease
FDA Expands GSK’s Arexvy Approval to Younger Adults at Elevated RSV Risk
As reported on PharmaBiz, the US Food and Drug Administration has broadened the approved use of GSK’s respiratory syncytial virus (RSV) vaccine, Arexvy, to include
Niemann-Pick Type C Disease
An Excerpt from “A Ribbon for Your Hair”
Editor’s Note: The following in from author Stephen Policoff, whose book A Ribbon for Your Hair is out today! Introduction When Stephen Policoff’s adopted daughter
Glioma
Telix Pharmaceuticals Advances Brain Cancer Imaging with European Regulatory Submission
A New Solution to Address Critical Gaps in Glioma Diagnosis and Management Telix Pharmaceuticals, a biopharmaceutical company specializing in therapeutic and diagnostic radiopharmaceuticals, has taken
Wet macular degeneration
VIS-101 Demonstrates Favorable Safety and Early Efficacy in Wet AMD
As reported on Healio, a novel biologic targeting two key angiogenic pathways has shown encouraging early results for the treatment of wet age-related macular degeneration
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Today is International Pompe Day, a day dedicated to raising awareness about Pompe disease — a rare genetic condition that affects how the body breaks down glycogen, leading to progressive muscle weakness and other serious health complications.
For many people living with Pompe disease, the journey can involve years of searching for answers, navigating complex symptoms, and learning to adapt to a life with a rare condition.
Today we recognize the strength of individuals and families affected by Pompe disease, the clinicians and researchers working to better understand it, and the communities that continue to advocate for awareness, earlier diagnosis, and improved care.
Awareness helps lead to understanding. Understanding helps lead to progress.
#InternationalPompeDay #PompeDiseaseAwareness #raredisease #rarediseaseawareness #PatientWorthy
Living with Spinal Muscular Atrophy (SMA) comes with experiences that are deeply personal, meaningful, and powerful. By sharing your journey, you have the opportunity to help others feel seen, understood, and less alone.
Patient Worthy is looking for individuals living with SMA to share their stories. Your voice may offer comfort to someone newly diagnosed, encouragement to a caregiver, or hope to another patient navigating similar challenges.
There is no right or wrong story to tell. Every experience matters. Whether your journey feels big or small, it has the power to make a difference.
If you are open to sharing your journey to help others along theirs, we would be honored to hear from you. To share your journey, click the link below.
bit.ly/4dV7gru
#spinalmuscularatrophy #sma #shareyourstory #PatientWorthy
Triple Negative Breast Cancer: A Breakthrough Treatment bit.ly/4txJybj Read now at PatientWorthy.com
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