C3 Glomerulopathy
FDA Approved Therapy for Rare and Severe Kidney Disease for Children Twelve or Older
In recent reports on MSN and AdventLS, it was shared that, as a result of the pioneering efforts of Dr. Carla Nester and researchers at
Sonelokimab Shows Sustained One-Year Benefits in Hidradenitis Suppurativa, With Encouraging Early Results in Adolescents
Good News for Lung Cancer Patients
Refuse to Disappear
LISTEN TO OUR PODCAST
THE LATEST FROM PATIENT WORTHY
Rare Disease
Authentic Data is Becoming an Asset Class
Our friends and contributors at the TREND Community have provided us with another enlightening article, this time about high-quality human-generated data becoming a scarce and
Alpha-1 antitrypsin deficiency
Grifols Starts Phase 3 Trial for a New, More Convenient Alpha-1 Treatment
What Happened? The healthcare company Grifols announced in a recent press release that it has enrolled and dosed the very first patient in a new
Rare Disease
Filling a Critical Gap: How TAF Removes Financial Barriers to Treatment
Editor’s Note: Patient Worthy is proud and honored to share this article, provided by our friends at The Assistance Fund. Every year, thousands of families
Rare Disease
FDA Extends Review of Praxis Epilepsy Drug Candidate Relutrigine by Three Months
As reported on BioSpace, Praxis Precision Medicines has received a three-month extension to the U.S. Food and Drug Administration’s review of relutrigine, its investigational therapy
Pancreatic Cancer
Investigational Tango–Revolution Drug Combination Shows Encouraging Early Activity in Pancreatic Cancer
Early Clinical Results Highlight Potential New Treatment Strategy As reported on STAT News, a novel investigational drug combination developed through a collaboration between Tango Therapeutics
Psoriasis
FDA Expands Risankizumab Approval to Pediatric Patients With Plaque Psoriasis and Psoriatic Arthritis
As reported on Becker’s Hospital Review, the FDA has approved risankizumab (Skyrizi) for use in children and adolescents aged 6 years and older with moderate-to-severe
Ebola
Clinical Trial to Evaluate Experimental Treatments Amid Expanding Bundibugyo Ebola Outbreak
According to STAT News, a major international clinical trial is about to begin in the Democratic Republic of the Congo (DRC) as health authorities respond
Thyroid eye disease
FDA Approves Viridian’s Lumvoa for Thyroid Eye Disease
As reported on PharmaBiz, the U.S. Food and Drug Administration (FDA) has approved Lumvoa (veligrotug‑vvze), a new therapy developed by Viridian Therapeutics, for the treatment
Cancer
Lilly Reports Phase 3 Success for Pirtobrutinib Combination in Relapsed CLL
As reported by PharmaBiz, Eli Lilly and Company has announced positive findings from its Phase 3 BRUIN CLL-322 trial evaluating pirtobrutinib (Jaypirca), a next-generation non-covalent
Alzheimer's disease
UAE Launches First Genomics-Led Trial Aimed at Preventing Alzheimer’s Disease
As reported on PharmaTimes, the United Arab Emirates has taken a significant step toward preventive neurology with the initiation of its first clinical trial guided
Obesity
FDA to Evaluate Sandoz’s Generic Tirzepatide Candidates
As reported by Yahoo Finance, the U.S. Food and Drug Administration (FDA) has accepted for review two applications from Sandoz for generic versions of tirzepatide,
Rare Disease
FDA Launches PreCheck Pilot to Accelerate U.S. Drug Manufacturing Capacity
As reported on FiercePharma, the U.S. Food and Drug Administration (FDA) has selected seven pharmaceutical and biotechnology companies to participate in its newly established PreCheck
Psoriasis
FDA Expands Approval of Roflumilast Cream (Zoryve) to Younger Children with Plaque Psoriasis
As reported on Healio, the U.S. Food and Drug Administration has broadened the indication for roflumilast cream 0.3% (Zoryve, Arcutis), allowing its use in children
Triple-negative breast cancer
“I Won’t Let This Define Me.”
Editor’s Note: Patient Worthy is honored to share this story from our friends at Elephants & Tea, originally written by Amy Hoffmann — a survivor
Dystonia
When Your Brain Won’t Quiet Down: A Look at Dystonia and Anxiety
One of the hardest things about living with dystonia is trying to explain what it feels like on the inside. People often see (not always for some) the muscle
COVID-19
The Future of Disease Understanding Starts with Listening
Editor’s Note: Patient Worthy is honored to share this article, provided by our friends at the TREND Community. To see the article in its original
Hidradenitis suppurativa
ICYMI: Make HStory Platform Offers Hidradenitis Suppurativa Education and Community Support
Editor’s Note: This article was originally published by Patient Worthy on 6/7/24, and is being republished due to our ongoing efforts to raise awareness of
FEATURED
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
@SepsisCanada Read Now!An international panel of 68 experts has released updated guidelines (2026) for managing sepsis and septic shock in pediatric cases.
Highlights of the Surviving Sepsis Campaign International Guidelines for the Management of Sepsis and Septic Shock in Children 2026 include:
✅ Start broad-spectrum antibiotics as soon as sepsis is recognized, ideally within 1 hour for septic shock. This is one of the most consistently supported interventions, with clear evidence that delays directly increase mortality.
✅ When fluids are not enough to restore blood pressure, norepinephrine is preferred over other agents due to stronger evidence for effectiveness and a safer side-effect profile.
✅ Identify and eliminate the source of infection (e.g., drainage of an abscess, removal of infected devices) as early as possible. This is a cornerstone of treatment with strong evidence linking delays to worse outcomes.
✅ In patients with sepsis-induced respiratory failure, use low tidal volume ventilation (~6 mL/kg). This strategy is backed by high-quality evidence showing a significant reduction in mortality.
Sepsis remains one of the leading causes of death in children worldwide. These guidelines help standardize care across hospitals and countries, giving clinicians a clearer, evidence-based roadmap.
Full guidelines:
link.springer.com/article/10.1007/s00134-026-08360-2
A Small Win I'm Celebrating Today Is...
When you're living with a rare or chronic illness, even the smallest victories can be worth celebrating. Some days, a win might look like getting out of bed, making it through a challenging appointment, taking a walk, managing symptoms, or simply finding a reason to smile.
No achievement is too small when you're navigating a health journey that requires strength, resilience, and courage every single day.
Today, we're celebrating the wins—big and small—that remind us of how far we've come.
💬 Share yours in the comments below and let's encourage one another. Your victory may be exactly the inspiration someone else needs today.
#raredisease #ChronicIllness #patientadvocacy #rarediseaseawareness #chronicillnesswarrior #PatientVoice #invisibleillness #Resilience #hope #smallwins #StrongerTogether #communitysupport #PatientWorthy
We've all been there. You tell yourself you'll make the appointment after the long weekend... and suddenly it's time to follow through.
If this feels a little too relatable, consider this your friendly reminder to make that call. Your future self will thank you.
#patientlife #WellnessReminder #PatientWorthy
