Patient Worthy

Hepatitis E

RNA-Targeting CRISPR: A Breakthrough Approach to Inhibiting Hepatitis E Virus

Bree Clare - May 26, 2026

Hemophilia

European Commission Expands Approval of Pfizer’s Marstacimab for Hemophilia Patients With Inhibitors

Metabolic dysfunction-associated steatohepatitis

Madrigal Highlights Expanding Evidence for Resmetirom in MASH at EASL 2026

Huntington's Disease

Breaking Through: UniQure’s Huntington’s Gene Therapy Pursues UK Approval After US Setback

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Generalized arterial calcification of infancy-1 (GACI1)

Sir’Savion’s Story

Editor’s Note: Patient Worthy is honored to share this article from our friends at the Brighter Hope Foundation. To see the article in its original

Ovarian Cancer

Labcorp Brings Breakthrough Diagnostic to Identify Platinum-Resistant Ovarian Cancer Patients

Labcorp announced the nationwide availability of Agilent Technologies’ PD-L1 IHC 22C3 pharmDx, the only FDA-approved companion diagnostic designed to identify patients with platinum-resistant ovarian cancer

Pancreatic Cancer

Daraxonrasib Delivers Landmark Survival Gains in Previously Treated Metastatic Pancreatic Cancer

As reported on drugs.com, Revolution Medicines has reported practice-changing efficacy results from a pivotal Phase 3 trial evaluating daraxonrasib in patients with metastatic pancreatic ductal

Primary Biliary Cholangitis

The PBCers Conference

This, year the PCBers will meet in Indianapolis, Indiana. They will be celebrating 30 years as a community, as well as celebrating a much brighter

Autoimmune hemolytic anemia

FDA Priority Review Accelerates Path for IMAAVY as First Treatment for Warm Autoimmune Hemolytic Anemia

Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review to IMAAVY® (nipocalimab-aahu), marking a significant milestone as the

Atopic dermatitis

Enveda’s Oral Candidate ENV-294 Shows Early Promise in Atopic Dermatitis

As reported on Business Wire, Enveda, a clinical-stage biotechnology company focused on discovering novel small-molecule medicines, has reported encouraging results from a Phase 1b study

Lupus

Insights into Gazyva as a Treatment for Lupus/SLE

A recent article from Inside Precision Medicine states: For decades, systemic lupus erythematosus (SLE) has resisted tidy solutions, a shapeshifting autoimmune disease that crosses into

Chronic obstructive pulmonary disease (COPD)

AstraZeneca’s Tozorakimab Shows Promise as First-in-Class COPD Breakthrough

AstraZeneca has announced positive results from the pivotal phase III MIRANDA trial of tozorakimab, a groundbreaking first-in-class monoclonal antibody designed to treat Chronic Obstructive Pulmonary

Astrocytoma

Listening to the Quiet Signs: A Twelve-Year Journey to Answers

On January 20, 2014, at 54 years old, I experienced what I casually called an “anxiety numbness event.” My left thumb and index finger went

HIV/AIDS

Merck’s Idvynso: A New Single-Tablet Breakthrough in HIV Treatment

The US Food and Drug Administration (FDA) has approved Idvynso, a groundbreaking two-drug single-tablet regimen combining 100 mg doravirine and 0.25 mg islatravir, for treating

Hepatic Adenoma

Against All Odds: Madison’s Victory Against a Hepatic Adenoma

My story began very suddenly on May 4th, 2025, when I was home from graduate school visiting my family. It had been a great day,

Glioblastoma

Experimental Ultrasound-Based Therapy Shows Early Promise Against Glioblastoma

Glioblastoma multiforme (GBM) is the most common malignant brain tumor in adults and remains one of the most difficult cancers to treat. Characterized by aggressive

Rare Disease

Maddie and the Drug that Turned Her Life Around

WebMD published a story about a young woman, Maddie, who had spent nine years in wheelchair confinement resulting from a rare metabolic disease related to

Cystinosis

Game-Changing Eye Drop Therapy Now Available to Canadian Cystinosis Patients

Health Canada has officially approved Cysklar® (cysteamine ophthalmic solution) 0.44%, marking a significant breakthrough for Canadians living with cystinosis. According to BusinessWire.com, the approval enables

Cervical Dystonia

Health Benefits of Putting Feelings to Words

Putting feelings into words does more than help you reflect. Brain imaging research shows it can shift activity inside emotional circuits. If you are someone

Rare Disease

“I Am More Than the Person in the Bed” – Kristian’s Message to the World

Most people see the bed first. That’s what defines me to them. The bed in the living room, the fact that I’m always in it.

Chronic urticarias

Enanta Begins First-in-Human Trial of Oral KIT Inhibitor for Chronic Urticaria

As reported by Business Wire, Enanta Pharmaceuticals has initiated its first clinical study of EDP-978, a novel oral inhibitor of the KIT receptor tyrosine kinase