Acromegaly
Tessa’s Journey with Acromegaly
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
Aardvark Pauses Late‑Stage Prader–Willi Trial After Cardiac Signals Emerge
- March 6, 2026
Myth or Truth? Avoid Soy If You Have a Hormone-Positive Cancer
Breaking New Ground: FDA Green-Lights Novel Breast Cancer Treatment Targeting Treatment-Resistant Tumors
Vico Therapeutics Begins Twice‑Yearly Dosing of VO659 in Phase 1/2 Trial for Huntington’s Disease and Spinocerebellar Ataxias
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THE LATEST FROM PATIENT WORTHY
Charcot-Marie-Tooth disease
Can the Cost of Having a Rare Disease Be Calculated?
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
IQIRVO: A Revolutionary PPAR Agonist Transforms Second-Line Treatment for Primary Biliary Cholangitis
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
Myelofibrosis
We Will Never Ring the Bell – My Myelofibrosis Story
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
Graves' Disease
What Functional Medicine and Holistic Care Taught Me About Healing Graves and TED
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
ADHD
Beyond the Diagnosis: Otsuka’s “All of ADHD” Campaign Reframes Healthcare Provider Perspectives on a Complex Condition
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
Activated PI3K delta syndrome
When Two Rare Diseases Collide: What My Son Taught Me About Immune System Complexity
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
Nipah Virus
Who is at Risk of Getting the Deadly Nipah Virus?
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
Narcolepsy
FDA Grants Priority Review to Takeda’s Oveporexton: A Potential Game-Changer for Narcolepsy Type 1 Treatment
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
Metabolic dysfunction-associated steatohepatitis
CHMP Backs Novo Nordisk’s Semaglutide for MASH, Opening Door to EU Approval
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
Graves' Disease
Rock Bottom in Mexico: The Thyroid Eye Disease Flare That Forced Me to Rebuild My Health
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
Duchenne Muscular Dystrophy
Aucta Pharmaceuticals Launches PYQUVI, Expands Branded‑Generic Portfolio in Rare Disease Care
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
Duchenne Muscular Dystrophy
CureDuchenne’s 2026 Schedule
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
Eye Disease
Faricimab Delivers Durable, Real-World Benefits for Retinal Vein Occlusion
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
ADHD
FDA Prioritizes Review of Otsuka’s Centanafadine for ADHD Treatment
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
Graves' Disease
The Emotional Toll of Thyroid Eye Disease: What Patients Are Not Told
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
Obesity
Dual Targeting Psoriatic Arthritis and Obesity: Taltz–Zepbound Combo Sets New Benchmark in TOGETHER-PsA Trial
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
Follicular lymphoma
Triplet Therapy Strategies Signal a Shift in Relapsed Follicular Lymphoma Care
As reported by Bioengineer, new insights published by Nastoupil L.J. in Nature Reviews Clinical Oncology (2026) highlight an important evolution in the management of relapsed
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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
Patient Worthy is thrilled to share this video from Prxtrialport!
"Each story is different, but the message is collective: we walk different roads, yet we walk together. Every patient’s health journey is shaped by personal experience, cultural context, and healthcare systems, yet beneath those differences lie common threads that unite us."
#ClinicalTrials #RareDisease #PatientWorthy
