Rare Disease
A Glance at 2025-2026 Flu Seasons
According to the CDC, a look back at April 2026 cumulative reports showed more than 139 pediatric deaths with approximately 250,000 people hospitalized. In simple
Resilience During Adversity
From Denial to Balance: My Journey with Lupus
Ultrasounds and Pain
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McCune-Albright Syndrome
Breakthrough Recognition: Atossa’s (Z)-Endoxifen Earns FDA Rare Pediatric Disease Designation for McCune-Albright Syndrome
Atossa Therapeutics Inc. has achieved a significant regulatory milestone with the U.S. Food and Drug Administration’s grant of Rare Pediatric Disease (RPD) designation to (Z)-endoxifen
Acute Myeloid Leukemia
New Hopes for Acute Myeloid Leukemia
An article from University of Michigan highlighted Jolanta Grembecka and her husband Tomasz Cierpicki, both assistant professors in U of M’s Department of Pathology, who
Ehlers-Danlos Syndrome
Buy Art. Fund Science. Change Lives
Milenia Makani is an abstract artist living in London. She lives with Hypermobile Ehlers-Danlos Syndrome (hEDS), and has launched an international art fundraiser to support
Cancer
When Breakthrough Drugs Need a New Home: Inside the Arvinas-Rigel Partnership
What happens when a pharmaceutical first-mover discovers its historic achievement may not be a blockbuster? Arvinas and Pfizer found an answer this week by handing
Parkinson's Disease
Claudine’s Journey with Parkinson’s Disease
What is Parkinson’s Disease – known as PD for short? Parkinson’s Disease was first discovered in 1817 by a British doctor named James Parkinson. From
Bipolar disorder
The Prevalence of Tick Infections in Pediatric Bipolar Disorders
A recent article by Dr. Rosalie Greenberg explores the possible relationship of infections caused by ticks and psychiatric conditions in children, including PANS or PANDA
Epidermolysis Bullosa
Expanding Hope for Rare Skin Disease: CHOP Becomes Sixth Treatment Center for Revolutionary ZEVASKYN Gene Therapy
Abeona Therapeutics Inc. has announced a significant expansion in access to ZEVASKYN, marking the activation of Children’s Hospital of Philadelphia (CHOP) as the sixth Qualified
Duchenne Muscular Dystrophy
Regenxbio Reports Positive Pivotal Data for Gene Therapy RGX-202 in Duchenne Muscular Dystrophy
As reported on PharmaBiz, Regenxbio has announced encouraging topline findings from the pivotal Phase III portion of its ongoing AFFINITY DUCHENNE clinical program evaluating RGX-202,
Sickle Cell Disease
Canadian Biotech Biossil Identifying Potentially Overlooked Drug Candidates
Biossil, based in Toronto and co-founded by Dr. Alexander Mosa (Chair, CSO and co-founder) and Anthony Mouchantaf (CEO and co-founder), is making headway for rare
Rare Disease
Poolbeg Pharma Expands Global IP Position with Canadian Patent for POLB 001 in CRS
As reported on PharmaTimes, Poolbeg Pharma has secured a Canadian patent protecting the use of p38 MAPK inhibitors, including its lead candidate POLB 001, for preventing
Rare Disease
How to Advocate for Yourself During Appointments
Editor’s Note: This story was originally published on Linked-In and is shared with permission by Olga Lucia Torres. She waited an hour and fifteen minutes to
Rare Disease
GLP-1–Based Therapies Linked to Reduced Risk of Sleep Apnea in Obesity
As reported on Healio, emerging real-world evidence suggests that glucagon-like peptide-1 (GLP-1) receptor agonists—and related dual agonists—may play a significant role in lowering the risk
Leukodystrophy
Connor’s Leukodystrophy Story
I remember the day I realized something was wrong, as clearly as if it happened this past weekend. It was a beautiful morning in June.
Lupus
The Quiet Revolution: When Hospitals Stop Being Your Second Home
Systemic lupus erythematosus punishes patients twice. First comes the disease itself, an autoimmune disorder where the body attacks its own tissues, causing debilitating fatigue, joint
Cholangiocarcinoma
FDA Expands Approval of Bizengri for Ultra-Rare Cholangiocarcinoma Subset
As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has approved Partner Therapeutics’ bispecific antibody Bizengri for adults with cholangiocarcinoma harboring an NRG1
Rare Disease
In Rare Disease Care, Coordination Should Be Common
Editor’s Note: Patient Worthy is honored to share this submission from Katie DiLorenzo, SVP, Patient and Pharmacy Services, PANTHERx® Rare. If you’re living with a
Epidermolysis Bullosa
Solving Cell Therapy’s Dirty Secret: How One Partnership Could Unlock Consistent Treatments
Mesenchymal stromal cells (MSCs) have tantalized the medical world for years. These versatile immune-modulating cells offer genuine hope for diseases ranging from epidermolysis bullosa, a
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Living with COPD can be challenging, but effective management goes beyond medication alone. From pulmonary rehabilitation and oxygen therapy to lifestyle changes and preventive care, a comprehensive treatment plan can help improve breathing, increase independence, and enhance quality of life.
Understanding available treatment options empowers individuals to take an active role in their care and work toward better respiratory health—one breath at a time. 💙🫁
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#copd #COPDawareness #PatientWorthy
To anyone living with a rare or chronic illness: Having to explain your condition over and over doesn’t make you “difficult”—it speaks to your resilience. It shows the strength it takes to advocate for yourself, to be heard, and to keep going in ways most people don’t see. Even on the days when you’re exhausted from repeating your story, your experience is valid. The emotional labor behind this journey isn’t talked about enough, and this is for everyone who’s had to explain their condition one too many times. 💙 #ChronicIllnessAwareness #RareDisease #InvisibleIllness #PatientWorthy #Support #Compassion #Empathy
Appointments can feel overwhelming, but sometimes the smallest preparations can make the biggest difference. Writing down questions, tracking symptoms, bringing a support person, or simply giving yourself time to decompress afterward can help make each visit a little more manageable. Every appointment is a step toward understanding, advocating for, and caring for your health. And that matters!
What is one thing that helps make appointments easier for you?
#patientadvocacy #healthtips #PatientWorthy
