Rare Disease
After the Diagnosis: Self-Harming Thoughts and So Many Questions
After being diagnosed with a rare disease or any other chronic health condition (in my case I have dystonia) and learning it is not a
Serina Therapeutics Begins First-in-Patient Dosing in Phase 1b Trial of SER‑252 for Advanced Parkinson’s Disease
- March 10, 2026
New Gene Editing Program Aims to Address Root Cause of PKU
From Psychiatric Bind to Clinical Solution: The VMAT2 Revolution in Treating Tardive Dyskinesia
Brain Cancers: FDA Approves Pembrolizumab Combo
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Graves' Disease
Thriving Is a Choice: How Community, Boundaries, and Mindfulness Saved My Life & Thyroid
Editor’s Note: Patient Worthy is proud to share part 10 of 10 of Elena Genik’s blog, detailing her journey with Graves disease and thyroid eye
ornithine transcarbamylase deficiency
Pruitt’s Legacy: Raising Awareness of OTC Deficiency and Genetic Testing
Rare Disease Month is deeply personal to our family. Before Pruitt, our youngest son, we had never heard of Ornithine Transcarbamylase (OTC) Deficiency, nor did
Stiff Person Syndrome
Celine Dion’s Stiff Person Syndrome Treatment and Progress
Ever since Celine Dion, the popular superstar, announced the cancellation of her 2023 tour due to a diagnosis of Stiff Person Syndrome, or SPS, the
Rare Disease
FDA Advances Rivoceranib-Camrelizumab Combination: A Promising Breakthrough for First-Line Unresectable Hepatocellular Carcinoma Treatment
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
Rare Disease
Nutcracker Syndrome, May-Thurner Syndrome, and More: Tytina’s Journey to a Proper Diagnosis
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
Graves' Disease
Why Graves and Thyroid Eye Disease Are Immune System Disorders, Not Thyroid Disorders
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
Melanoma
FDA Listens: When Regulatory Bodies Recognize Humanity in the Fight Against Advanced Melanoma
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
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
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
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