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
Aucta Pharmaceuticals Launches PYQUVI, Expands Branded‑Generic Portfolio in Rare Disease Care
- February 13, 2026CureDuchenne’s 2026 Schedule
Faricimab Delivers Durable, Real-World Benefits for Retinal Vein Occlusion
FDA Prioritizes Review of Otsuka’s Centanafadine for ADHD Treatment
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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
Obesity
Roche’s Obesity Drug Shows Promise Against Lilly and Novo Nordisk
Roche has announced encouraging results from its Phase II trial of CT-388, an experimental obesity treatment that achieved placebo-adjusted weight loss of 22.5% over 48
Squamous-cell carcinoma
EMA Endorses First Immunotherapy Option for Advanced Anal Cancer
The European Medicines Agency (EMA) has issued a positive opinion recommending that retifanlimab (Zynyz) be approved for an expanded indication to treat adults with advanced
Graves' Disease
The Invisible Battle No One Warned Me About: Developing Thyroid Eye Disease
Editor’s Note: Patient Worthy is honored to share part 3 in an ongoing 10-part series, written and shared with us by Elena Genik. When My
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Yellow for Yiannis #RareDiseaseRare disease day may only be one day a year but for us it is EVERY SINGLE DAY.
Rare disease day is a global awareness 🦓🧬 held annually on the last day of February. Its purpose is to shine a light on the challenges faced by individuals living with rare diseases like Yiannis and to encourage international advocacy, research, and support.
With 70% of rare diseases starting in childhood, this initiative underscores the importance of collective action in improving outcomes and providing emotional and medical assistance to affected families which through Yellow for Yiannis we are able to accomplish.
Along with funding critical research to develop a viable treatment for ALL with IRF2BPL-NEDAMSS once and for all.
Thank you BFFS & BABES For creating these incredible shirts to help us bring awareness to IRF2BPL-NEDAMSS—for us to share our stripes in solidarity for rare disease month!!
If you can please purchase a shirt or anything from our magical collection each purchase supports our critical research efforts!! 🧬🦓 If able please donate here through this link to help us reach our goals!!
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Thanks everyone, it truly takes a village. Thanks for being part of ours!
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#itp Platelet Disorder Support Association“I think sometimes in life you look for something you want to dedicate to a cause that’s a little bit bigger than yourself.” -Derek Zimmerman
Ever wonder what PDSA’s Board of Directors actually does? Tune into our latest podcast episode featuring the incoming and outgoing Board Chairs, Derek Zimmerman and Peter Pruitt. Listen now at pdsa.org/podcast, Apple and Spotify. #ITP #ITPresources #ITPpodcast #PDSA
