Leukemia
Where the Pain Used to Be
Editor’s Note: Patient Worthy is honored to share this article from our friends at Elephants & Tea, originally written by Kiki Khan, a leukemia survivor.
Vera’s Trutakna Shows Signs of Preserving Kidney Function in IgA Nephropathy, Challenging Voyxact’s Differentiation
- August 25, 2026
New Consensus Recommendations Aim to Strengthen Communication and Decision-Making in Neuroblastoma Care
Investigational Therapy Shows Promise for Guillain-Barré Syndrome in Late-Stage Testing
FDA Approves First Gene Therapy for Rare Inherited Deafness, Offering New Hope for Children
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Rare Disease
Jon’s Caregiver Story
Editor’s Note: This post was written and submitted to us by Jon Scheinman, and originally shared on LinkedIn. To see the article in its original
Diabetes Type 2
Lilly’s Retatrutide Achieves Strong Weight Loss and Glycemic Benefits in Two Phase 3 Obesity Studies
As reported on Drugs, Eli Lilly has reported positive topline findings from two additional Phase 3 trials of retatrutide, its investigational triple hormone receptor agonist
Asthma
AstraZeneca’s Triple-Inhaler Asthma Therapy Moves Closer to EU Approval
As reported on PharmaBiz, AstraZeneca has reached an important regulatory milestone in Europe after the European Medicines Agency’s Committee for Medicinal Products for Human Use
Breast Cancer
EMA Backs Enhertu Plus Pertuzumab as Potential New First-Line Standard for HER2-Positive Metastatic Breast Cancer
As reported on PharmaBiz, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation supporting approval of AstraZeneca
Lupus
Roche Ends Lupus T-Cell Engager Program but Maintains Focus on Autoimmune Cell Therapies
As reported on Fierce BioTech, Roche has discontinued development of its investigational T-cell engager (TCE) RG6382 for systemic lupus erythematosus (SLE), following an internal review
HIV/AIDS
Novel HIV Vaccine Generates Strong Broadly Neutralizing Antibody Responses in Preclinical Study
Researchers from La Jolla Institute for Immunology (LJI), Scripps Research, and IAVI have reported a major advance in HIV vaccine development, demonstrating an experimental vaccine
Coats Disease
An Eye-Opening Look into Coats’ Disease Research
Coats disease, is a rare retina condition diagnosed primarily in children. Symptoms may include, eye drift, blurry vision, the appearance of a white or silver
Triple-negative breast cancer
CHMP Backs Datroway for First-Line Treatment of Metastatic Triple-Negative Breast Cancer in EU
As reported on Stock Titan, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of
Melanoma
FDA Review Raises Questions About Replimune’s Melanoma Therapy Ahead of Advisory Committee Vote
As reported on Reuters, Replimune’s experimental cancer treatment RP1 is facing increased regulatory scrutiny after U.S. Food and Drug Administration (FDA) reviewers expressed concerns about
Hepatocellular Carcinoma
Cheryl’s Liver Cancer Story
In my younger years, beginning around the age of 30, I was living fast and careless. I was the party girl, always drinking, never thinking
Rare Disease
FDA Clears Kenvue’s OTC Tylenol-Naproxen Combination for Long-Lasting Pain Relief
As reported on Pharmaceutical Technology, Kenvue has received US Food and Drug Administration (FDA) approval for an over-the-counter (OTC) pain medication that combines acetaminophen and
Prostate Cancer
CRISPR-Based RNA Editing May Boost Immunotherapy Response in Prostate Cancer
As reported on Science Daily, a novel CRISPR-driven RNA editing strategy could help overcome one of the biggest challenges in prostate cancer treatment: resistance to
Breast Cancer
EU Approves Enhertu as First Tumor-Agnostic HER2-Targeted Antibody-Drug Conjugate
As reported on Stock Titan, the European Commission has approved Enhertu® (trastuzumab deruxtecan) as a treatment for adults with previously treated, unresectable or metastatic HER2-positive
Cervical Dystonia
The Quiet Anger: Processing the Emotional Toll of Chronic Health Conditions
Living with chronic pain or other health conditions is, in many ways, an exercise in perpetual, involuntary mindfulness. For those of us navigating long-term conditions—like
Rare Disease
FDA Clears TorEx Lung Perfusion System, Expanding Options for Donor Lung Assessment
As reported on TechTimes, the U.S. Food and Drug Administration (FDA) has granted Premarket Approval to the TorEx® Lung Perfusion System, a new ex vivo
Rare Disease
FDA Issues Final Guidance for Psychedelic Drug Trials, Clarifying Expectations for Developers
As reported on Clinical Trials Arena, the US Food and Drug Administration (FDA) has released its final guidance on the clinical development of psychedelic therapies,
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Baltimore, Maryland, Nov. 10-14, @NationalSocietyofGenetic CounselorsYour professional growth doesn’t stop when you leave the classroom.
At #NSGC26, explore how new evidence and insights are changing genetic counseling practice, with fresh topics including:
🧬Use of AI for gene curation and variant classification
🧬Emerging approaches to genome sequencing in adult neurology
🧬A genetic counselor’s role in multi-cancer early detection
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Guillain-Barré syndrome (GBS) can develop quickly, often following an infection, and its symptoms can look different from person to person.
Learning more about rare conditions like GBS can help encourage awareness, understanding, and recognition of what patients may experience throughout diagnosis, treatment, and recovery.
Share this post to help more people learn about GBS! If you'd like to share your story, click here: ow.ly/JKT250ZEWEk or the link in our bio to learn more.
#GuillainBarreSyndrome #GBS #rarediseaseawareness #autoimmunedisease #shareyourstory #PatientWorthy
