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
Promising New Parkinson’s Treatment Shows Encouraging Early Results
- September 14, 2026
How Research Transformed Cystinosis: From a Fatal Childhood Disease to a Treatable Lifelong Condition
Raising Awareness of Hunter Syndrome: Stephanie’s Caregiver Story
Imfinzi–Imdelltra Combination Extends Survival in First-Line Extensive-Stage Small Cell Lung Cancer
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THE LATEST FROM PATIENT WORTHY
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
Rare Disease
TAVR Is Reaching Younger Patients. What Happens Decades Later?
Editor’s Note: Patient Worthy is honored to share this article, originally written by David St. Denis, President & Director at Anteris Technologies. Transcatheter aortic valve
Huntington's disease
The Researcher Who Would Not Be Tested
Although Nancy Wexler’s efforts were largely responsible for creating a genetic test to locate the Huntington’s gene, she refused to take the test herself saying
Breast Cancer
Guardant360 CDx Approved as Companion Diagnostic for AstraZeneca’s Etcamah in ESR1-Mutated Breast Cancer
As reported on Fierce BioTech, Guardant Health has secured U.S. Food and Drug Administration approval for its Guardant360 CDx liquid biopsy test as the companion
Rare Disease
Dual-Drug Strategy Shows Promise Against Treatment-Resistant Prostate Cancer
As reported on Science Daily, researchers at the University of Michigan have identified a potential new therapeutic approach for aggressive prostate cancers that become resistant
Multiple Myeloma
BMS Achieves Milestone with Next-Generation CAR-T Therapy for Multiply-Treated Myeloma Patients
GPRC5D-Targeting Approach Positions Company to Expand Post-BCMA Treatment Options As reported on Fierce BioTech, Bristol Myers Squibb announced positive results from its pivotal Quintessential trial
Rare Disease
To Stand Beside Suffering
Editor’s Note: We’re honored to share the following article from our friends at the Courageous Parents Network. To see this article in its original format,
Pompe Disease
Machine Learning Maps and Discovers Clinical Endpoints in Pompe Disease Using Real-World Data
Editor’s Note: Patient Worthy is honored to share this article from Rareatives, originally written by our friend Jessica Lynn. To see the article in its
Colorectal cancer
When the Silence Got Loud
Editor’s Note: Patient Worthy is honored to share this story from our friends at Elephants & Tea, originally written by Bill Phillips, a colorectal cancer
PTEN Hamartoma Tumor Syndrome
New PTEN Research Could Help Predict Cancer Risk in Rare Genetic Syndrome
A single genetic change can have dramatically different effects from one person to the next. That is the reality for people living with PTEN hamartoma
Diabetes Type 2
FDA Expands Mounjaro’s Label to Include Cardiovascular Risk Reduction in Type 2 Diabetes
As reported on QZ, the U.S. Food and Drug Administration has approved a new indication for Eli Lilly’s tirzepatide (Mounjaro), allowing the therapy to be
Polycythemia vera
FDA Clears Takeda’s Mimrylo for Polycythemia Vera, Introducing a Novel Treatment Option
As reported on MedCity News, the U.S. Food and Drug Administration has approved Takeda Pharmaceutical’s Mimrylo (rusfertide) for the treatment of erythrocytosis in adults with
Colorectal cancer
BioNTech Ends Phase 2 Trial of Personalized mRNA Therapy in High-Risk Colorectal Cancer
As reported on Drugs, BioNTech has decided to halt development of its Phase 2 BNT122-01 study investigating autogene cevumeran (BNT122/RO7198457) in patients with resected colorectal
Gastroesophageal junction carcinoma
Phase 3 HERIZON-GEA-01 Analysis Strengthens Survival Evidence for Zanidatamab-Based Regimens in HER2-Positive Gastroesophageal Cancer
As reported on PharmaBiz, BeOne Medicines has reported positive results from the second interim analysis of the phase 3 HERIZON-GEA-01 trial, further supporting the use
Leukemia
Think Beyond Remission to Protect the Life We Saved: A Young Man’s Journey with T-cell Acute Lymphoblastic Leukemia/Lymphoma
At 22, a young person’s world should be filled with possibilities—a career to build, places to travel, friendships to make, and a future to imagine.
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Not what we have, but what we enjoy, constitutes our abundance. John Petit-Senn
GLUT1 Deficiency Foundation THIS IS TONIGHT!!🔔 Reminder: GLUT1 Gathering tomorrow!
We invite Parents/Caregivers to learn about Getting Connected with Sports, featuring several GLUT1 moms sharing their experiences. All details below!
Let us know your favorite sport in the comments 👇
September GLUT1 Gathering for Parents/Caregivers:
🎤 Special Guests: Leslie Holleman, Erin Meisner, Debbie Stoddard
🗓️ Monday, September 14th: 8 PM ET, 7 PM CT, 6 PM MT, 5 PM PT
🔗 Check your Family Network email or the GLUT1 Community Connect Facebook group for the link to register.
#GLUT1Deficiency #GLUT1 #GLUT1Gatherings @followers
#braintumor Head For The Cure Cancer CommonsThe Rare Enough podcast from Head For The Cure (HFTC) features conversations that highlight the strength and stories of the brain tumor community.
In Episode 30, host DJ Stewart—GBM survivor and HFTC Community Outreach Coordinator—welcomes Unsha Bakker, MSN, RN, OCN, CBCN, Brain Cancer Support and Solutions Alliance (BCSSA) Nurse Navigator at Cancer Commons.
Together, they discuss the vital role of nurse navigation in helping patients and families find clarity, support, and individualized care throughout the brain cancer journey.
Watch here on YouTube—bit.ly/CCRareEnoughPodcast30—or listen on your favorite podcast platform!
It's a wonderful way to honor National Glioblastoma Awareness Day!
#RareEnough #BrainTumorAwareness #NurseNavigator #BrainCancerSupport #CancerCommons #HeadForTheCure #BCSSA #BTSM #GlioblastomaAwarenessDay
