Patient Worthy

LISTEN TO OUR PODCAST

THE LATEST FROM PATIENT WORTHY

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

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