Duchenne Muscular Dystrophy
Duchenne Families Made the Sacrifices Asked. FDA Withheld the Hope.
Editor’s Note: This story was submitted to us by Angelina Olivera, the mom of a 14-year-old with Duchenne muscular dystrophy, and the sister of two
Stanford Medicine Achieves First Clinical Use of Compact Upright Proton Therapy
- June 10, 2026
“Hello, My Name Is”
MediciNova Advances Intellectual Property for Glioblastoma Combination Therapy
Otsuka’s Voyxact Advances IgA Nephropathy Treatment Landscape with Phase 3 Data
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Breast Cancer
FDA Extends Review of AstraZeneca’s Investigational Breast Cancer Therapy
As reported in BioPharmaDive, the U.S. Food and Drug Administration (FDA) has postponed its decision on camizestrant, an experimental oral therapy for breast cancer being
Hypertrophic Cardiomyopathy
HAYA Therapeutics Advances First-in-Class RNA Therapy Into Phase 1 for Cardiac Fibrosis in nHCM
As reported on PharmaBiz, HAYA Therapeutics has reached an early clinical milestone with its lead investigational therapy, HTX-001, announcing that the first cohort of participants
Rare Disease
The Child Who Defied the Textbooks
The human body is governed by an ancient, rapid-response defense system. When an invader attacks, the innate immune system rushes to the front lines like
Hodgkin Lymphoma
Dear Cancer, Three Years Have Gone By
Editor’s Note: This story was originally written by Julia Alessandrine—a survivor of Hodgkins Lymphoma—and shared with us by our friends at Elephants & Tea. To
Rare Disease
The Heart Behind Every Clinical Trial
Editor’s Note: This article was submitted to us by Regina Portnoy. A clinical trial may be remembered for a breakthrough treatment, a new therapy, or
Cervical Dystonia
Resilience During Adversity
Having lived with chronic pain and involuntary muscle contractions from a neurological movement disorder called dystonia since 2001, I felt defeated many times. But I
Lupus
From Denial to Balance: My Journey with Lupus
Looking back, the signs were there long before I was diagnosed with lupus. As a child, I struggled with gastrointestinal issues and a variety of
Rare Disease
Ultrasounds and Pain
Imagine being able to turn down the dial on severe pain without taking a single pill or undergoing surgery. Scientists in the UK have just
mastocytosis
A Systemic Mastocytosis Journey: From Diagnosis to Advocacy
Editor’s Note: Patient Worthy is honored to present this story, shared with us by our friends at The Mast Cell Disease Society. To see the
Pancreatic Cancer
Daraxonrasib Is Promising. But Here’s What Pancreatic Cancer Patients Need to Know First
By Dr. Avishek Kumar When someone hears there may finally be a breakthrough for pancreatic cancer, the natural reaction is hope. In clinic, however, I
Rare Disease
Heart Attacks and Your Brain
1. A Physical Smoking Gun in the Heart-Brain Connection When people experience cognitive problems, mood drops, or chronic anxiety after surviving a heart attack, the
Bronchiectasis
Infex Therapeutics Reports Positive Phase IIa Data for RESP‑X in Bronchiectasis Patients with Pseudomonas Colonisation
As reported on PharmaBiz, Infex Therapeutics has announced encouraging results from a phase IIa clinical study of RESP‑X, an investigational monoclonal antibody being developed for
ADHD
Cingulate Receives FDA Complete Response Letter for ADHD Candidate Over Manufacturing Issues
As reported on FiercePharma, Cingulate Therapeutics has encountered a regulatory setback in its effort to bring its lead attention-deficit/hyperactivity disorder (ADHD) therapy to market, after
MPS III (Sanfilippo Syndrome)
Should vs. Want: Guiding Insights from a Palliative Care Clinician
Editor’s Note: This article was originally written by Joanne Huff, and shared with us by our friends at the Courageous Parents Network. To see the
Alpha-1 antitrypsin deficiency
Efdoralprin Alfa Shows Superior Efficacy Over Standard Augmentation Therapy in AATD Emphysema
As reported on PharmaBiz, new findings from the phase 2 ElevAATe trial suggest that the investigational agent efdoralprin alfa may represent a meaningful advance in
COVID-19
FDA Clears First Oral Medication for Post-Exposure COVID-19 Prevention
As reported on Beckers Hospital Review, the U.S. Food and Drug Administration has authorized ensitrelvir (brand name Xocova) as the first oral antiviral indicated for
Rare Disease
Hidden for Six Weeks: How My Brain Injury Revealed Itself
To this day, I still don’t remember the car accident that changed my life. It was Thursday, August 8, 2024 — a day like any
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Have you been impacted by Multifocal Motor Neuropathy (MMN)? Your experience matters. Patient Worthy is looking for people affected by MMN to share their perspective and help make patient voices heard. By sharing your story, you can help raise awareness, inspire understanding, and make a difference for the community.
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#multifocalmotorneuropathy #mmn #shareyourstory #PatientWorthy
Multifocal motor neuropathy (MMN) is a rare neurological condition that can cause progressive muscle weakness and impact everyday activities. Because MMN is often misunderstood or mistaken for other conditions, awareness and education are important.
Swipe to learn more about MMN. Share your story with us here: bit.ly/4dV7gru or click the link in our bio.
#multifocalmotorneuropathy #mmn #shareyourstory #PatientWorthy
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