Rare Disease
The Weight of a Decision
Editor’s Note: We’re proud to be the first to share this story by Blair Young Whitworth, a Parent Champion of the Courageous Parents Network. There
Unlocking a New Era: Illumina’s 5-Base Solution Bridges Genomics and Epigenomics for Multiomic Discovery
- October 27, 2025
“The Aftermath: Nobody Told Me This Part Would Hurt Too: – Megan’s Fight Against Non-Hodgkin’s Lymphoma
“My Own Worst Enemy” – Anna’s Experience with Hodgkin Lymphoma
Narcolepsy Linked to Higher Cardiovascular Disease Risk: Study Highlights Need for Regular Screening
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THE LATEST FROM PATIENT WORTHY
Macular degeneration
Sanofi’s Gene Therapy for Wet AMD Earns FDA Fast Track Designation
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SAR402663, Sanofi’s investigational gene therapy for neovascular age-related macular degeneration (AMD). As
Rare Disease
Brewing Good Health: New Research Reveals Coffee’s Powerful Gut Benefits
Coffee lovers have long celebrated their favorite beverage for its taste and energy boost, but recent research offers another compelling reason to savor that daily
Lupus
Rady’s Lupus Journey: Carried by Faith, Fueled by Hope
I came to the United States from the Philippines in June 2016, full of excitement and dreams. I was ready to build a new life,
Huntington's disease
Experimental Gene Therapy Slows Huntington’s Disease Progression in Landmark Trial
An experimental gene therapy known as AMT-130 has become the first treatment to successfully slow the progression of Huntington’s disease, marking a potential breakthrough in
Friedreich's Ataxia
FDA Signals Flexibility as Lexeo’s Gene Therapy for Rare Disease Advances Toward Faster Approval
Lexeo Therapeutics announced a significant regulatory development this week: the U.S. Food and Drug Administration (FDA) is open to a faster approval pathway for the
Glanzmann thrombasthenia
Sherry Crutchfield’s Journey with Glanzmann Thrombasthenia
Editor’s Note: Patient Worthy is proud to share this story from our friends at the Glanzmann’s Research Foundation (GRF). To see the article in its
Metabolic dysfunction-associated steatohepatitis
MASH (formerly known as NASH) Patient Stories Needed
Your story has the power to educate, motivate, and make a real difference in the lives of others. We are looking for patients with Metabolic
Metabolic dysfunction-associated steatohepatitis
MASH (formerly known as NASH) Patient Stories Needed
Your story has the power to educate, motivate, and make a real difference in the lives of others. We are looking for patients with Metabolic
Parkinson's Disease
Unlocking the Future: Sleep Disorder Biomarkers Predict Parkinson’s and Dementia
In a groundbreaking international effort led by Dr. Shady Rahayel of Université de Montréal and reported by MSN.com, researchers have discovered new ways to predict
Breast Cancer
Datroway Shows Survival Benefit in Late-Stage Trial for Triple-Negative Breast Cancer
AstraZeneca and Daiichi Sankyo have announced promising Phase 3 trial results for Datroway, their investigational antibody-drug conjugate (ADC) targeting triple-negative breast cancer (TNBC). The trial
Parkinson's Disease
Investigational Cell Therapy Bemdaneprocel Shows Sustained Safety and Promising Motor Improvements at 36 Months in Parkinson’s Disease Trial
Bayer AG and its subsidiary BlueRock Therapeutics LP have announced encouraging 36-month follow-up data from the Phase I exPDite clinical trial of bemdaneprocel (BRT-DA01), an
Glanzmann thrombasthenia
Vincenzo’s Journey with Glanzmann Thrombasthenia
Editor’s Note: Patient Worthy is proud to bring you this story from our friends at the Glanzmann Research Foundation. To see the article in its
Rare Disease
Beyond the Parachute: Why Patient Recruitment Partners Belong in the Cockpit
Clinical trials often begin with optimism, sites are launched, projections look promising, and timelines are set. But when enrollment stalls, sponsors frequently call-in patient recruitment
Glanzmann thrombasthenia
Jyll’s Journey with Glanzmann Thrombasthenia
Editor’s Note: Patient Worthy is honored to share this story from our friends at the GRF (Glanzman’s Research Foundation). To see the article in its
Parkinson's Disease
Unlocking the Brain: CU Anschutz Uses Neuropixels Technology to Illuminate Parkinson’s Disease
Doctors and researchers at the University of Colorado Anschutz Medical Campus have achieved a groundbreaking milestone by employing advanced Neuropixels technology in the study of
Rare Disease
Redefining Autoimmune Care: T Cell Engagers and the Promise of Immune Reset
Autoimmune diseases affect an estimated 400 to 600 million people globally, posing a growing challenge for healthcare systems and patients alike. Traditionally, treatment has focused
Lupus
My Diagnosis Didn’t Silence Me, It Lit a Fire
Editor’s Note: Patient Worthy is proud to bring you this story-share, originally written by Gwen Pike, founder of the podcast Empowerment in Illness. When I was
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🧠 October is Pituitary Awareness Month, a time to shine a light on one of the body’s most powerful but often overlooked glands.
The pituitary gland plays a vital role in regulating hormones that affect growth, metabolism, and overall health. Yet, disorders like Cushing’s disease, acromegaly, and hypopituitarism are frequently misdiagnosed or misunderstood.
This month, we honor the strength of those living with pituitary conditions and advocate for earlier diagnosis, better treatment, and stronger support systems.
💙 Join us in raising awareness by sharing your story or tagging someone who inspires you.
#PituitaryAwarenessMonth #RareButStrong #PatientVoicesMatter #PatientWorthy
Unlocking a New Era: Illumina’s 5-Base Solution Bridges Genomics and Epigenomics for Multiomic Discovery bit.ly/4hf3hI9. Read now at PatientWorthy.com
#PatientWorthy #BloodTesting
Transitioning to adult care #neurodevelopmentaldisorders
