Rare Disease
You Do Not Need to Be Healed to Be Held: Love and Chronic Illness
Have you ever looked in the mirror and wondered, “Who am I now? Why did this diagnosis happen to me? When will this be over?
Novartis Suspends Eight CAR-T Studies After Patient Deaths Trigger Safety Review
- September 3, 2026
Cherry Angiomas May Represent an Underrecognised Vascular Feature of Neurofibromatosis Type 1
BioCryst Wins Japanese Approval for Pediatric Use of ORLADEYO in Hereditary Angioedema
New Brush Test Could Make Oral Cancer Detection Easier
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THE LATEST FROM PATIENT WORTHY
Dermatomyositis
Roivant and Priovant Secure FDA Approval for First Targeted Oral Therapy for Dermatomyositis
As reported on Pharma Phorum, Roivant Sciences and its subsidiary Priovant Therapeutics have received U.S. Food and Drug Administration (FDA) approval for Lisraya (brepocitinib), marking
non-small cell lung cancer
AstraZeneca Secures Global Rights to EGFR-Targeted Lung Cancer Therapy ZEGFROVY
As reported on Business Wire, AstraZeneca has finalized its previously announced licensing agreement with Dizal Pharmaceutical, gaining exclusive worldwide rights to develop and market ZEGFROVY
Rare Disease
Basking Biosciences formulates Reversable Clot Busting Therapy for Acute Stroke
Treating acute ischemic strokes involves a precarious clinical balance: physicians must dissolve arterial clots to save brain tissue without triggering catastrophic intracranial hemorrhaging. To address
SYNGAP1
Lorelei, the SYNGAP1 Warrior
Editor’s Note: Patient Worthy is honored to share the following article, originally published by our friends at CURE SYNGAP1. To see the article in its
Rare Disease
From Ocean to Operating Room: How Fish Skin Is Quietly Revolutionizing Wound Healing in America
A Sustainable Biologic Solution Gains Major Insurance Coverage and Proves Superior to Synthetic Alternatives in Real-World Clinical Data In the world of advanced wound care
Cancer
AIVITA Biomedical Supports Development of Personalized Dendritic Cell Cancer Vaccine Platform
As reported on PharmaBiz, a new study published in Cell Stem Cell highlights a promising approach to cancer immunotherapy that combines stem cell technology with
Rare Disease
Moderna Strengthens Cancer Pipeline as FDA Authorizes Updated COVID-19 Vaccines
As reported on BioPharmaDive, Moderna captured attention this week with two major developments: a multibillion-dollar financing initiative aimed at expanding its oncology portfolio and regulatory
Dermatomyositis
Orsini Selected as Specialty Pharmacy Partner for Newly Approved Dermatomyositis Therapy LISRAYA
As reported on PR NewsWire, Orsini has been named a specialty pharmacy partner for Priovant’s LISRAYA™ (brepocitinib), a once-daily oral treatment recently approved by the
Hereditary ATTR Amyloidosis
CARDIO-TTRansform: Eplontersen Fails to Improve Cardiovascular Outcomes When Added to Standard Therapy in ATTR-CM
As reported on Healio, the phase 3 CARDIO-TTRansform study, the largest clinical trial conducted to date in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM), found
Rare Disease
When Other People Doubt Your Body Long Enough, You Start Doing It for Them
For a long time, I knew something was wrong. And then, when whatever had been building inside my body finally came to the surface, it
Pancreatic Cancer
A Breakthrough 50 Years in the Making: FDA Approves First Drug to Directly Target Pancreatic Cancer’s Primary Driver
RASONQUE Represents a Watershed Moment for a Disease That Has Resisted Treatment Since RAS Was Discovered as a Cancer-Causing Gene Pancreatic cancer is among the
Autoimmune hemolytic anemia
When Your Body’s Immune System Attacks Your Blood: FDA Approves First Treatment for Warm Autoimmune Hemolytic Anemia
A Rare Disease Gets Its First FDA-Approved Medicine—And It Works Differently Than Anything Available Before Imagine your immune system getting the signals completely wrong. Instead
Rare Disease
“Failing First” Means Your Insurance Watches You Get Worse Before Helping You Get Better
Twenty-plus pills a day. Two biologic injections a month, two more every week. That’s my baseline just to function like a “normie” human and keep
ADHD
More Than Medication: How Emotional Support Animals and Nutrition Play a Hidden Role in Managing ADHD
Editor’s Note: Patient Worthy is honored to share this article, originally written by Danviel Villarte . Danviel is part of the Outreach Team at Receptive,
AADC Deficiency
The Facebook Post That Changed Our Daughter’s Life
Editor’s Note: Patient Worthy is honored to share this story, submitted to us by Richard Poulin III. Late one night, a seemingly ordinary Facebook post
Rare Disease
Beyond the Price Tag: What Generic Drug Tariffs Could Mean for Patients’ Access to Medication
Editor’s Note: Patient Worthy is honored to share this article, originally written by Renzo Luzzatti, President and CEO of US-Rx Care. The White House recently
Rare Disease
FDA Announces Nationwide Recall of Certain Thyroid Medication Due to Excess Potency Risk
As reported on The Hill, Vitruvias Therapeutics has initiated a voluntary nationwide recall of a specific lot of thyroid medication after testing indicated the tablets
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UPCOMING EVENTS
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Understanding the symptoms and causes of Hereditary Angioedema (HAE) can help patients and families feel more informed, prepared, and empowered. While every HAE journey is different, knowledge can be a powerful tool for navigating challenges and advocating for the care you deserve. Click the link below to share your story with us today!
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#HAE #HereditaryAngioedema #raredisease #PatientWorthy #shareyourstory
Not every condition progresses quickly or causes obvious symptoms right away. For some patients, changes can happen gradually over months or even years, making them easier to overlook or harder to connect. This can add another layer of complexity to diagnosis, monitoring, and understanding how a condition may change over time.
#rarewordoftheweek #raredisease #rarediseaseawareness #PatientWorthy
Sometimes, the most ordinary things become quiet witnesses to the biggest parts of our story.
A pill organizer might look like seven small compartments, but for someone living with a chronic or rare disease, it can represent years of medication changes, new routines, trial and error, small victories, and difficult days.
There are the medications that became part of everyday life, the ones that didn’t work, the doses that changed, and the moments when taking another pill became so routine that you barely thought about it.
Because sometimes, the objects we use every day carry pieces of our story that no one else can see. 🤍 #MedicationJourney #ChronicIllnessAwareness #PatientPerspective #PatientWorthy
