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
Experimental Gene Therapy Slows Huntington’s Disease Progression in Landmark Trial
FDA Signals Flexibility as Lexeo’s Gene Therapy for Rare Disease Advances Toward Faster Approval
Sherry Crutchfield’s Journey with Glanzmann Thrombasthenia
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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
Parkinson's Disease
Study Links Age to Increased Alzheimer’s-Related Brain Changes in Parkinson’s Patients Without Dementia
A new study published in Aging-US reveals that Parkinson’s disease (PD) patients diagnosed in their 80s are significantly more likely to exhibit amyloid-beta buildup, which
Progressive Familial Intrahepatic Cholestasis
A New Era for PFIC Patients in Japan: Bylvay® Receives Landmark Approval
In a significant advancement for rare disease treatment in Japan, the Ministry of Health, Labour and Welfare (MHLW) has approved Bylvay® (odevixibat) for the management
Breast Cancer
Finding Strength in Connection: How Breast Cancer Support Communities Are Transforming Patient Care
When a woman hears the words, “You have cancer,” her world shifts in an instant. The journey ahead—marked by treatment, uncertainty, and emotional upheaval—can feel
Barth Syndrome
Good News for an Ultra-Rare Disease
Each year, Patient Worthy covers the American College of Medical Genetics conference, the leading source of both genetic education for health care professionals and a
Rare Disease
FDA Decisions Could Reshape Pharma Landscape in Late 2025
As 2025 winds down, the pharmaceutical industry is bracing for a series of pivotal FDA decisions that could redefine treatment paradigms and reshape competitive dynamics
Breast Cancer
Digital Innovation Transforms Breast Cancer Care: NCCN Launches Interactive Guidelines Navigator
In a landmark step for personalized cancer care, the National Comprehensive Cancer Network® (NCCN®) has launched the NCCN Guidelines Navigator™—a cutting-edge digital platform that redefines
Brugada syndrome
Living with Brugada Syndrome
Editor’s Note: Patient Worthy is honored to bring you this patient story-share from Michael Grivas. For more information and resources, please visit the SADS Foundation.
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A new experimental gene therapy seems to show promising results in trials, slowing the progression of Huntington's disease. Read more here:
patientworthy.com/2025/10/17/experimental-gene-therapy-slows-huntingtons-disease-progression-in-l...
It’s #FactFriday! Hereditary Angioedema (HAE) is a rare, inherited disease that can cause sudden, severe swelling. Learn these 5 key facts and help raise awareness! #HAEAwareness #RareDisease #HereditaryAngioedema #HAE #PatientWorthy
#ShareYourStory with us here: bit.ly/4dV7gru
#functionalneurologicaldisorders📢 NICE has just published NG252, a new guideline on Rehabilitation for Chronic Neurological Disorders, which includes Functional Neurological Disorder (FND).
Read the full guideline here: www.nice.org.uk/guidance/ng252
The guideline highlights:
*Comprehensive assessment for diverse symptoms.
*Integrated care plans across healthcare professionals.
*Support for daily living including education and employment.
*Focus on mental wellbeing.
This comes on the heels of NHS England recognising FND as a core neurology subspecialty: www.england.nhs.uk/publication/specialised-neurology-services-adults/
Another big step in giving FND the visibility, recognition, and specialist care it deserves. Now we need services to be accessible for all. 💜
#fnd #fndawareness #functionalneurologicaldisorder #fndaction
