porphyria
Her Final Year: What Love Looked Like When We Didn’t Have Answers
Editor’s Note: Patient Worthy is honored to present this story by Heather Doyle, originally published by the United Porphyrias Association. To see the article in
European Commission Expands Approval of Pfizer’s Marstacimab for Hemophilia Patients With Inhibitors
- May 25, 2026
Madrigal Highlights Expanding Evidence for Resmetirom in MASH at EASL 2026
Breaking Through: UniQure’s Huntington’s Gene Therapy Pursues UK Approval After US Setback
Guardant Health Secures FDA Clearance for Expanded Liquid Biopsy Test
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Breast Cancer
Beyond Your Grip
Editor’s Note: This piece, originally written by Kayla DeSonier, was shared with us by our friends at Elephants & Tea. To see the article in
Neurofibromatosis Type 2
The Silent Struggle – Mental Wellness in NF2 Surgery
Editor’s Note: This article was shared with us by our friends at the Cure NF2 Foundation, in honor of May 22nd being World NF2 Awareness
Colorectal cancer
Merck and Exelixis Team Up to Transform Treatment for High-Risk Colorectal Cancer Patients
Exelixis has taken a major step forward in its colorectal cancer development pipeline by securing supplies of Merck’s Keytruda for an upcoming phase 3 clinical
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy: Coping and Caring
Betty Vertin is a mother, writer and a fierce defender of the decisions she and her husband Jason made about the seven children they brought
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
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
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Throughout the week, Patient Worthy is highlighting Huntington's Disease with educational content aimed at raising awareness and fostering greater understanding in our community.
We’ll be sharing information on our socials and bit.ly/425L89T to educate, empower, and support those affected.
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Breaking Through: UniQure's Huntington's Gene Therapy Pursues UK Approval After US Setback bit.ly/4f1KjWo Read now at PatientWorthy.com
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