Esophageal Cancer
Stephen’s Journey with Esophageal Cancer
My name is Stephen Savage. I’m happy to say I’m a cancer survivor. I was diagnosed in February, 2019 with a form of adenocarcinoma: esophageal
First Patient in the World Treated With Gene Therapy for Cockayne Syndrome
- August 12, 2026
Qiagen Secures FDA Clearance for First QIAstat-Dx Bloodstream Infection Test in the US
England to Add Spinal Muscular Atrophy to Newborn Screening Program in 2027
Otsuka Reports Two-Year Kidney Function Benefit With Sibeprenlimab in Phase 3 IgA Nephropathy Trial
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Epidermolysis Bullosa
Health Canada Prioritizes Review of FILSUVEZ for EB-Related Wounds
As reported of The Manila Times, Health Canada has granted priority review to Chiesi Global Rare Diseases’ new drug submission for FILSUVEZ® (birch triterpenes) topical
Polycythemia vera
Silence Therapeutics Advances Divesiran After Positive Phase 2 Results in Polycythemia Vera
As reported on Fierce Biotech, Silence Therapeutics reported positive phase 2 findings for divesiran, an investigational small interfering RNA (siRNA) treatment for polycythemia vera (PV),
Spinal Muscular Atrophy
Scholar Rock’s SMA Therapy Application Advances Despite Manufacturing Site Change
As reported on Drugs, Scholar Rock says the US Food and Drug Administration’s review of its biologics license application for apitegromab, an investigational treatment for
Pemphigus vulgaris
From Breakdown to Breakthrough – Humberto’s Pemphigus Vulgaris Story
Last year, I came down with a severe autoimmune disorder. It came out of nowhere. And it was bad. I was hospitalized three times over
SYNGAP1
Porter’s Perspective | A SYNGAP1 Sibling
Editor’s Note: We are honored to share this story from our friends at CURE SYNGAP. To see this article in its original format, please click
Multiple Sclerosis
From Diagnosis to Purpose: Nick’s MS Journey
It was 2013, I was 21 years old, studying abroad in Italy, living the semester every college student dreams about. Then I woke up one
Tardive Dyskinesia
Teva Data Suggest Sustained Benefits of Austedo in Tardive Dyskinesia Management
New research presented by Teva Pharmaceuticals at Psych Congress Elevate 2026, and reported on PharmaBiz, adds to the growing body of evidence supporting the use
Cervical Dystonia
The Oversensitized Nervous System: Why Symptoms Fluctuate
Living with dystonia, or any chronic condition, places the nervous system under continuous pressure and strain. When symptoms are unpredictable or uncomfortable, the brain learns to
Bladder cancer
Novel Immunotherapy Offers Bladder Cancer Patients a Path Beyond Surgery
New Treatment Strategy May Eliminate Need for Immediate Organ Removal As reported on Bioengineer, groundbreaking clinical investigation led by researchers at Mayo Clinic suggests that
Cancer
The Cancer Dietitian’s Chocolate Avocado Pudding
Editor’s Note: The following recipe was written and shared with us by our friend Julie, the Cancer Dietitian. I cannot tell you how many people
Uveitis
Emerging Evidence: Common Vaccines May Shield Against Eye Inflammation
New Research Reveals Protective Link Between Routine Immunizations and Uveitis Prevention A comprehensive analysis of patient health records reported on Medscape has uncovered a promising
Idiopathic intracranial hypertension
Daniel’s CSF Shunt for Idiopathic Intracranial Hypertension
Editor’s Note: The following patient story was submitted to us by Daniel S.—an animal lover, sports fan, and former IIH patient. When you think about
Lupus
Belimumab Dose Spacing May Be an Option for Selected Patients With SLE in Sustained Remission
As reported on MedScape, gradually extending the interval between belimumab doses may allow carefully selected patients with systemic lupus erythematosus (SLE) in long-term remission to
Primary Biliary Cholangitis
FDA Grants Breakthrough Therapy Designation to Pemafibrate for Primary Biliary Cholangitis
As reported on PR Newswire, the US Food and Drug Administration has granted Breakthrough Therapy designation to Kowa’s investigational therapy K-808 (pemafibrate) for primary biliary
Rare Disease
Enfortumab Vedotin/Pembrolizumab Regimen Outperforms Neoadjuvant Chemotherapy in Resectable MIBC
As reported on The ASCO Post, perioperative treatment with enfortumab vedotin-ejfv plus pembrolizumab resulted in superior event-free survival, overall survival, and pathologic response rates compared
Ebola
Ebola Outbreak and Lessons Learned
As reported by the BBC, the Ebola outbreak in DR Congo was declared in May 2026 and is now the second worst on record with
Mast Cell Activation Syndrome
How to Improve the Chances of Getting Useful MCAS Test Results
Editor’s Note: The following article was originally written by Dr. Linda Bluestein. Linda Bluestein, MD, is a board-certified anesthesiologist and internationally recognized expert in hypermobility
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We replay our mistakes, criticize our appearance, compare our journey to someone else’s, and forget to recognize how far we’ve come.
Give yourself the grace you so freely give to others. You don’t have to be perfect to be worthy of love, kindness, or acceptance. And you don’t have to earn the right to appreciate the person looking back at you in the mirror.
Be a little gentler with yourself today. You’re doing better than you think. 🤍
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Rare disease patients don't just live through unanswered questions.
They help answer them.
Through their experiences, advocacy, and resilience, they are helping shape the future of research, treatment, and care for generations to come.
The future of medicine is being written by patients, too.
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