Elevar Therapeutics has achieved a significant regulatory milestone with the US Food and Drug Administration’s acceptance of a resubmitted New Drug Application for rivoceranib in
Sharing My Stripes 🦓 This Rare Disease Month, I’m honored to share my journey living as someone with a rare disease and what Rare Disease
Editor’s Note: Patient Worthy is honored to share part 9 of 10 of Elena Genik’s blog documenting her experiences with Graves disease and thyroid eye
When patients and families face a life-or-death disease, bureaucracy often feels like the enemy. Yet in early February 2026, the FDA delivered something rare: a
Hi! My name is Tessa, and I have a rare disease called Acromegaly. My journey started in 2015 when we discovered I had a 9x10x12
Editor’s Note: Patient Worthy is pleased to share this article, submitted to us by George Simpson, who volunteers for the CMT Research Foundation. There are
Primary biliary cholangitis (PBC) represents a chronic, progressive autoimmune liver disease characterized by the destruction of small bile ducts, leading to cholestasis and potentially fatal
Editor’s Note: The above photograph is credited to Patient Voice. “Ringing the bell” is an enduring image associated with cancer survivorship. It represents victory, celebration
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 and thyroid eye
Otsuka has launched an ambitious awareness initiative designed to fundamentally shift how healthcare professionals understand and approach attention-deficit/hyperactivity disorder (ADHD). As reported by FiercePharma.com, the
When my son was eight months old, he had already been hospitalized more times than I can count for infections that seemed far too severe
It took two new cases of the deadly Nipah virus to remind people in West Bengal, India of the 1998 outbreak that killed over one
Takeda Pharmaceutical has reached a significant milestone in the development of oveporexton (TAK-861), with the US Food and Drug Administration officially accepting and granting Priority
As reported on MedicalXpress, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for several therapies this
Editor’s Note: Patient Worthy is proud to share part 7 of 10 of Elena Genik’s blog series, detailing her experiences with Graves disease and thyroid
As reported on the Manila Times, Aucta Pharmaceuticals has officially introduced PYQUVI™ (deflazacort) oral suspension 22.75 mg/mL, marking the company’s entry into the U.S. commercial
CureDuchenne is hosting several free events in 2026 to support families living with Duchenne and Becker muscular dystrophy. These events, called CureDuchenne CARES, will take
Twelve‑month, real‑world data from a large U.S. registry suggest that faricimab provides effective, durable, and safe treatment for retinal vein occlusion (RVO), reinforcing findings from
We are excited to share that Patient Worthy is headed to Washington, DC for Rare Disease Week! 🦓 💙
During Rare Disease Week, advocates from across the country gather to amplify the voices of the rare community, share powerful stories, and push for policies that improve access, research, and care. We're honored to stand alongside patients, caregivers, and other organizations making a difference.
If you'll be in DC next week, let us know in the comments! Let's keep raising awareness and driving change, together!
#rarediseaseweek2026 #raredc2026 #PatientWorthy
Behind every rare diagnosis is a story that deserves understanding.
💬 What’s one thing you wish people truly understood about rare disease?
Drop your thoughts below! #RareDiseaseMonth #RareDiseaseAwareness #RareDisease #PatientWorthy #ReplyAll
During Rare Disease Month, Patient Worthy is honored to share a story from Steven who has been diagnosed with Waldenström's macroglobulinemia.
"My name is Steven and I am 73 years old, and in 2014, I was diagnosed with Waldenström's macroglobulinemia. Having a rare blood disease means staying on top of my health by constant follow-ups at a specialist several states away.
Having this rare disease means taking it one day at a time. I want to do things on my own, but I appreciate all of the help I get as my activities are limited at work.
I look forward to February each year as Rare Disease Month calls attention to those who have been diagnosed with a rare disease.
February is also another chance to advocate for rare disease as I hope one day there will be a cure for Waldenström's macroglobulinemia instead of just being treatable."
#RareDiseaseMonth #RareDisease #RareButNotAlone #WeCareAboutRare #ShareYourStripes #ShareYourStory #PatientWorthy
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