Rare Disease
The Value of Living with Tremendous Challenges
If you got past the title without rolling your eyes or getting angry (I understand if you did either or both as you will see
EMA Endorses First Immunotherapy Option for Advanced Anal Cancer
The Invisible Battle No One Warned Me About: Developing Thyroid Eye Disease
Artificial Intelligence Advances Eye Disease Diagnosis: Deep Learning Shows Promise in Distinguishing Thyroid Eye Disease from Orbital Myositis
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THE LATEST FROM PATIENT WORTHY
Pain Management
A Cellular Solution to America’s Opioid Crisis: Biotech Advances Alternative to Chronic Pain Management
Breaking the Cycle of Prescription Dependency The opioid epidemic that has devastated American communities for decades may finally have a novel challenger. According to Bezinga.com,
Fibromyalgia
Axsome Launches Phase III FORWARD Trial of AXS-14 for Fibromyalgia
As reported by Clinical Trials Arena, Axsome Therapeutics has begun dosing patients in its FORWARD Phase III clinical trial, a pivotal study designed to evaluate
Duchenne Muscular Dystrophy
Netflix Documentary “The Remarkable Life of Ibelin” Honored at 2026 EURORDIS Black Pearl Awards
Brussels — On February 24, 2026, the acclaimed Netflix documentary The Remarkable Life of Ibelin received the Media & Awareness Raising Award at the EURORDIS
Prostate Cancer
Breaking Down Healthcare Barriers: How One Man’s Struggle Opened Doors for Thousands with Prostate Cancer
The Three-Year Fight That Changed Everything When Giles Turner learned his postcode could determine whether he received life-saving cancer treatment, he didn’t accept it quietly.
Epidermolysis Bullosa
New Dedicated Clinical Trial Site Launched to Accelerate Epidermolysis Bullosa Research
As reported on PM Live, a new clinical trial hub focused on epidermolysis bullosa (EB) has been established at University Medical Center Groningen (UMCG) in
Biliary Atresia
Forged in Fire: My Journey with Biliary Atresia from 115 Pounds to a Life of Purpose
To the patient, the parent, the caregiver, and the person in the “limbo” of waiting: I am one of you. My name is Aaron Jackson,
IgA Nephropathy (IgAN)
Terminal Ileum’s Rogue B Cells: A Novel Gateway to IgA Nephropathy
A comprehensive multi-omics analysis has identified the terminal ileum as a critical source of pathogenic antibody-producing cells in IgA nephropathy (IgAN), one of the most
Hunter Syndrome
One-Time Gene Therapy Offers New Hope for Hunter Syndrome
As discussed in a recent interview between Krystal Jacques and Prof. Brian Bigger, a novel stem cell–based gene therapy is showing early promise as a
Achondroplasia
Oral FGFR Inhibitor Shows Promise for Children With Achondroplasia
As reported on MedPage Today, an oral targeted therapy may offer a safe and effective alternative to injectable treatment for children with achondroplasia, according to
Ataxia-telangiectasia
Research Opportunities in Ataxia and Movement Disorders
Editor’s Note: Patient Worthy is honored to share this post from Martha Harlam, Director of the Solution Project. Are you a student or researcher passionate
Dravet Syndrome
Choosing Joy: Erika’s Journey of Raising a Child with Dravet Syndrome, a Rare Form of Epilepsy
In the whirlwind of early motherhood, days blur together in a haze of feedings, diaper changes, and sleepless nights. You’re learning your baby’s every sound,
Breast Cancer
Revolutionary Sensor Detects Cancer Biomarkers at the Molecular Level Without Complex Fabrication
A radically new type of optical sensor can identify individual cancer-related proteins in blood, a capability that could revolutionize how early-stage breast cancer is diagnosed
non-small cell lung cancer
ImmunityBio Reports Encouraging Evidence That ANKTIVA Enhances Immune Recovery in NSCLC When Paired With Checkpoint Inhibitors
As reported on Business Wire, ImmunityBio has released new clinical findings suggesting that its interleukin‑15 superagonist, ANKTIVA® (nogapendekin alfa inbakicept), may help restore immune function
Rare Disease
FDA Approves First Treatment for Stem Cell Transplant–Associated Thrombotic Microangiopathy
The U.S. Food and Drug Administration (FDA) has approved Yartemlea (narsoplimab‑wuug), marking the first authorized therapy for hematopoietic stem cell transplant‑associated thrombotic microangiopathy (TA‑TMA) in
Hodgkin Lymphoma
Immunotherapy Breakthrough Offers Teens with Hodgkin Lymphoma a Better Path Forward
Young cancer patients with advanced Hodgkin lymphoma now have access to a significantly more effective treatment option that dramatically reduces their need for radiation therapy
ADHD
Landmark Review Identifies Most Effective Evidence‑Based Treatments for ADHD
As reported by Technology, a major evidence synthesis published in The BMJ has delivered the clearest picture to date of which therapies offer the strongest
Rare Disease
Pediatric Palliative Care
Editor’s Note: Patient Worthy is honored to bring you this article, originally written by Kelsey Stanczyk, and shared with us by our friends at the
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