Rare Disease
A Guide to Staying Well and Strong for New Caregivers
For new caregivers supporting a parent, partner, or relative at home, the days can turn into an unbroken string of decisions, vigilance, and worry. The
ASTRO Issues First-Ever Radiation Therapy Guideline for Bladder Cancer
- September 20, 2026
FDA Expands Kerendia Approval to Adults With CKD Linked to Type 1 Diabetes
Biomarkers
Planning Accessible Travel: What Actually Makes a Trip Work
LISTEN TO OUR PODCAST
THE LATEST FROM PATIENT WORTHY
Multiple Myeloma
An Unexpected Diagnosis Changed Everything: Otis’ Journey with Multiple Myeloma
When I was 39 years old, my life changed in an instant. I was a father of two young children, my daughter was six and
Idiopathic Pulmonary Fibrosis
Rentosertib Linked to Younger Biological Age Signatures in IPF Trial
As reported on News-Medical, an experimental treatment for idiopathic pulmonary fibrosis (IPF), rentosertib, was associated with shifts toward younger biological age profiles across multiple protein-based
Psoriasis
FDA Grants Priority Review to Takeda’s Psoriasis Candidate Zasocitinib
As reported on PharmaBiz, the U.S. Food and Drug Administration has accepted Takeda’s New Drug Application for the investigational oral therapy zasocitinib (TAK-279) and assigned
Gastroesophageal junction carcinoma
“You’re Stronger Than You Think You Are”
My name is Terri, and I live in the small town of Searcy, Arkansas, where I’ve proudly connected with countless people and their families over
Rare Disease
FDA Unveils Pilot Program Aimed at Accelerating Early-Stage Clinical Trials
As reported on Fierce Biotech, the U.S. Food and Drug Administration (FDA) has launched a new pilot program designed to streamline the investigational new drug
XLID98
The Thousand-and-Second Word: My Brother’s Life with XLID-98
Editor’s Note: This story was originally written and submitted to us by Anthony Clark. “Okay, Michael, hold the ball up to your ear, then throw
Dupuytren's Contracture
New Progress for Dupuytren’s Contracture
The condition Dupuytren disease, also called Dupuytren contracture, is a chronic disorder of connective tissue in the palm. Tissue gradually becomes thickened and tight, forming
Hepatocellular Carcinoma
FDA Greenlights Acepodia’s Investigational ADC Candidate ACE723 for Advanced Liver Cancer Trial
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has authorized Acepodia to proceed with clinical testing of ACE723, an investigational antibody-drug conjugate
Small cell lung cancer
Roche-Partnered B7-H3 ADC Meets Phase 3 Endpoint in Small Cell Lung Cancer, Shows Competitive Safety Profile
As reported on Fierce BioTech, a phase 3 study conducted by MediLink has delivered positive results for Roche-partnered antibody-drug conjugate (ADC) tambotatug pelitecan (tam-peli), also
Parkinson's Disease
Promising New Parkinson’s Treatment Shows Encouraging Early Results
What’s the Good News? Researchers have successfully completed initial testing of an experimental Parkinson’s medication called TT-P34. The early results are very encouraging and suggest
Cystinosis
How Research Transformed Cystinosis: From a Fatal Childhood Disease to a Treatable Lifelong Condition
The history of cystinosis is one of the most successful examples of rare disease research leading to meaningful treatments. In a recent interview with the
Hunter Syndrome
Raising Awareness of Hunter Syndrome: Stephanie’s Caregiver Story
This is my son Nikolai. Nikolai was recently diagnosed with Mucopolysaccharidosis type 2, or “Hunter Syndrome.” A rare genetic form of childhood dementia, it’s caused
Small cell lung cancer
Imfinzi–Imdelltra Combination Extends Survival in First-Line Extensive-Stage Small Cell Lung Cancer
As reported on PharmaBiz, a combination of Imfinzi (durvalumab) and Imdelltra (tarlatamab) has delivered significant improvements in survival outcomes for patients with extensive-stage small cell
Achalasia
“Lead the Way” with Dustin Chapman
At the age of 18, I was diagnosed with a rare swallowing disorder called Achalasia. At the time of this video, I had undergone several
Duchenne Muscular Dystrophy
A New App for Duchenne Muscular Dystrophy Caregivers
In honor of World Duchenne Awareness Day on September 7th, Mal Duffin has invited us to share this app designed for parents whose children are living
Pancreatic Cancer
FDA Authorizes Daraxonrasib for Metastatic Pancreatic Cancer After Positive Phase 3 Trial Results
As reported on World Pharma News, the U.S. Food and Drug Administration has granted approval to daraxonrasib, an oral RAS-targeting therapy, for adults with metastatic
Ebola
Ebola Continues To Be a Global Threat
A novel investigational Ebola vaccine has shown encouraging safety and immunogenicity results in a first-in-human clinical trial, supporting further development of an alternative vaccination strategy
FEATURED
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
Not every disease presents with a long list of recognizable symptoms. For some patients, having only a few signs can make it harder to see the full picture, especially when those symptoms could be explained by more common conditions. This can add another layer of complexity to the diagnostic journey and is an important reminder that the number of symptoms someone experiences doesn’t always reflect the significance of their condition.
#RareWordOfTheWeek #RareDisease #RareDiseaseAwareness #PatientWorthy
From our friends PTEN Hamartoma Tumor Syndrome Foundation
www.jaad.org
PTEN hamartoma tumor syndrome (PHTS) is an autosomal dominant disorder caused by pathogenic germline variants in the PTEN gene, characterized by macrocephaly, neurodevelopmental delays, and a high lif...
