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
The U.S. Food and Drug Administration has agreed to review Otsuka Pharmaceutical’s application for centanafadine, a new extended‑release therapy under investigation for attention‑deficit hyperactivity disorder
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
A first-of-its-kind Phase 3b study has shown that combining Eli Lilly’s Taltz (ixekizumab) with Zepbound (tirzepatide) delivers superior benefits for adults living with both active
We had the pleasure of meeting Krista in person last week at Rare Disease Week on Capitol Hill! Krista is one of ten 2025 Rare Artist Awardee winners. She is a talented wood-burning artist, advocate, wife, and mom of two. Krista was diagnosed with Usher syndrome, a rare genetic condition that affects her hearing and vision. Her vision loss, caused by retinitis pigmentosa, has narrowed her visual field from the typical 180° to just 5° of central vision. She wears hearing aids and uses a white cane daily to navigate independently!
“My art shares what it looks like to navigate life with dual sensory loss while still choosing creativity, connection, and joy. When I reflect on Rare Disease Month, it means visibility and research. It means community and advocacy. It means being seen, heard, and feeling worthy. I will continue to "wear my stripes" proudly!”
#RareDC2026 #RareDiseaseWeek #PatientWorthy #RareArtist
Getting a diagnosis for Huntington’s disease is rarely simple. It often involves a combination of neurological exams, medical history, genetic testing, and imaging to help doctors understand what’s happening in the brain.
For many families, the process can bring a mix of answers, uncertainty, and difficult decisions—especially when genetic testing is involved. Learning about diagnosis and testing is an important step toward understanding the condition, accessing care, and supporting those affected.
Awareness helps ensure people recognize the signs, seek appropriate evaluation, and receive the support they deserve. If you're interested in sharing your story, click here: bit.ly/4dV7gru
#HuntingtonsDisease #HuntingtonsDiseaseAwareness #ShareYourStory #PatientWorthy
March is Brain Injury Awareness Month. Today and every day, we stand with survivors, caregivers, and the countless individuals navigating life after a brain injury. You are seen, supported, and never alone 💚 #braininjury #BrainInjuryAwarenessMonth #MoreThanMyBrainInjury #BrainHealth
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