Duchenne Muscular Dystrophy
CureDuchenne’s 2026 Schedule
CureDuchenne is hosting several free events in 2026 to support families living with Duchenne and Becker muscular dystrophy. These events, called CureDuchenne CARES, will take
Redefining Cancer Treatment: Podcast Interview with Dr. Charles Link on Sync-T and the Future of Immunotherapy
- March 2, 2026
“At 44, I Thought I Was Starting Over” – Kristian’s Bladder Cancer Story
British Columbia Researchers Develop a System to Reduce Ovarian Cancer by 80%
Breakthrough in Rare Bone Disease: FDA Fast-Tracks Garetosmab for Fibrodysplasia Ossificans Progressiva
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THE LATEST FROM PATIENT WORTHY
Eye Disease
Faricimab Delivers Durable, Real-World Benefits for Retinal Vein Occlusion
Twelve‑month, real‑world data from a large U.S. registry suggest that faricimab provides effective, durable, and safe treatment for retinal vein occlusion (RVO), reinforcing findings from
ADHD
FDA Prioritizes Review of Otsuka’s Centanafadine for ADHD Treatment
The U.S. Food and Drug Administration has agreed to review Otsuka Pharmaceutical’s application for centanafadine, a new extended‑release therapy under investigation for attention‑deficit hyperactivity disorder
Graves' Disease
The Emotional Toll of Thyroid Eye Disease: What Patients Are Not Told
Editor’s Note: Patient Worthy is honored to share part 6 of 10 of Elena Genik’s series of blog posts detailing her journey with Graves disease
Obesity
Dual Targeting Psoriatic Arthritis and Obesity: Taltz–Zepbound Combo Sets New Benchmark in TOGETHER-PsA Trial
A first-of-its-kind Phase 3b study has shown that combining Eli Lilly’s Taltz (ixekizumab) with Zepbound (tirzepatide) delivers superior benefits for adults living with both active
Follicular lymphoma
Triplet Therapy Strategies Signal a Shift in Relapsed Follicular Lymphoma Care
As reported by Bioengineer, new insights published by Nastoupil L.J. in Nature Reviews Clinical Oncology (2026) highlight an important evolution in the management of relapsed
Myelofibrosis
How Anemia Could Signal a Rare Blood Cancer
Editor’s Note: Patient Worthy is honored to share this article from our friends at Heal Canada, written by Karen Hawthorne. Anemia is one of the
Eye Disease
Optic Nerve Stem Cells Redraw the Map of Retinal Vascular Repair
A new study in Nature Communications and reported by Scienmag.com overturns a central assumption in eye biology by showing that key stem cells for the
Triple-negative breast cancer
Datroway Receives U.S. Priority Review for First‑Line Treatment of Metastatic Triple‑Negative Breast Cancer
In a recent statement by AstraZeneca, AstraZeneca and Daiichi Sankyo’s antibody‑drug conjugate Datroway (datopotamab deruxtecan) has been granted Priority Review by the U.S. Food and
Graves' Disease
When “Normal Labs” Are Not Normal: What Thyroid Patients Need to Know
Editor’s Note: Patient Worthy is pleased to share part 5 of 10 in an ongoing series of blog posts, provided to us by Elena Genik.
Skin Disease
Swiss Biotechs Gain Momentum with FDA Fast Track Option for Palmoplantar Pustulosis
A trio of Swiss biotechnology companies is advancing diverse therapeutic pipelines, with recent developments spanning inflammatory skin disease, immune‑mediated hair loss, and hard‑to‑treat cancers. Together,
Propionic Acidemia
Moderna and Recordati Partner on mRNA‑3927 for Propionic Acidemia: Clinical Development Advances Toward Pivotal Readout
As reported on BioSpace, Moderna has entered a commercialization agreement with Recordati valued at up to $160 million to support late‑stage development and future market
Cancer
Nanoparticles Targeting Tumors Eliminates Most Chemotherapy Side Effects
Scientists find polymer-coated nanoparticles together with therapeutic drugs offer promise for cancer treatment, including treatment for ovarian cancer. According to Science Daily, these nanoparticles may
Eye Disease
Turning Heart Data Into an Early Warning System for Vision Loss
A UCLA Health study published on UCLAHealth.org suggests that numbers already used to gauge heart health may also offer an early warning for serious eye
Cancer
New Immunotherapy Strategy Targets Tumor ‘Bodyguards,’ Shows Powerful Results in Preclinical Cancer Models
As reported on ScienceDaily, a research team at the Icahn School of Medicine at Mount Sinai has unveiled a novel immunotherapy that tackles metastatic cancer
Graves' Disease
Medical Gaslighting Is Real: The Appointment That Changed Everything
Editor’s Note: We’re honored to share part 4 of 10 of an ongoing blog series, originally written by Elena Genik. When You Go to a
MPS I (Hurler Syndrome)
FDA Places Clinical Holds on REGENXBIO’s MPS Gene Therapy Programs After Tumor Detected in Trial Participant
As reported on drugs.com, REGENXBIO has disclosed that the U.S. Food and Drug Administration has halted clinical testing of its investigational gene therapies RGX‑111 and
Myelofibrosis
James’ Story: Navigating Life with Myelofibrosis
Editor’s Note: Patient Worthy is honored to share this patient story, provided to us by our friends at Heal Canada, and originally written for the
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Connect with stakeholders to create powerful partnerships, advance orphan therapeutics and inspire advocacy at the Rare Disease Summit, March 17-19 in Philadelphia, PA, US. You’ll generate solution-focused approaches surrounding patient access, reimbursement, commercialization, launch, partnering and more. Save 10%: 26PATWOR10. Register at bit.ly/4qrLAbc
Autoimmune AssociationToday, we kick off Autoimmune Awareness Month!
Awareness isn’t just noise or a ribbon or a hashtag. Awareness leads to earlier diagnoses, fewer years spent being dismissed, stronger research, better care, and lives changed because people are finally taken seriously.
This month, we’re raising awareness to drive real progress.
👉 Read why awareness matters—and how you can help. autoimmune.org/blog/autoimmune-awareness-month-why-awareness-matters/
This week on Patient Worthy, we're shining a spotlight on Huntington's Disease. All week long, we’ll be sharing meaningful insights information, and opening space for patients and caregivers to share their personal journeys. To learn more or join the conversation, visit patientworthy.com for the latest updates.
Have a story to share? We'd love to hear from you: bit.ly/4dV7gru
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