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
Breaking New Ground: Milsaperidone Offers Patients a Fresh Option for Serious Mental Illness
FDA Escalates Scrutiny of GLP‑1 Compounders as Industry Reports Key Clinical and Regulatory Updates
A New Shield Against Cancer: The Lynch Syndrome Vaccine
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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
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
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Connect with stakeholders to create powerful partnerships, advance orphan therapeutics and inspire advocacy at the Rare Disease Summit, March 17-19 in Philadelphia, PA, US. You’ll generate solution-focused approaches surrounding patient access, reimbursement, commercialization, launch, partnering and more. Save 10%: 26PATWOR10. Register at bit.ly/4qrLAbc
Meet Gabriela who is diagnosed with Lysinuric Protein Intolerance (LPI). Patient Worthy is honored to share her story!
"As a little girl, I thought miracles only happened in movies. I never imagined I would live one.
For years, I struggled with extreme exhaustion, vomiting, confusion, and behavioral episodes that were misdiagnosed. We saw multiple specialists searching for answers. In 2018, I underwent major spinal fusion surgery for a 60° scoliosis curve without even knowing I had an undiagnosed metabolic disorder that could have caused life-threatening complications during surgery. Looking back, I see God’s mercy covering me even before we had a diagnosis.
In April 2019, during a severe episode, one doctor decided to check my ammonia levels. They were over 500. That single test saved my life.
I was diagnosed at Nicklaus Children’s Hospital with Lysinuric Protein Intolerance (LPI), a rare metabolic disorder. Finally, we had answers.
Today, I’m 21 and studying Speech-Language Pathology and Communication at Nova Southeastern University. My pain became my purpose.
My hope in sharing this is bigger than awareness. I pray that ammonia testing becomes an emergency room protocol.
You are not your diagnosis.
And miracles are real."
#LysinuricProteinIntolerance #MetabolicDisorder #RareDisease #ShareYourStory #PatientWorthy
#lupus #car-T The New Yorker PresentsTalaya Reid’s teen years were marked by exhaustion, with fatigue so severe that she spent afternoons in bed. The cause of this lethargy was left undiscovered for years, until the summer of 2017, when Reid, then 21, noticed a rash on her face after a day at the beach. Her doctor dismissed it as nothing serious. But when the rash persisted she sought care from a dermatologist who raised a more ominous possibility: lupus. Blood tests confirmed the diagnosis. That was only the beginning of her journey.
After years of lupus flareups, Reid decided to try CAR-T therapy, a treatment previously reserved for certain blood cancers. CAR-T cells are a “living drug” that scientists create by extracting T cells from the patient’s immune system, genetically reprogramming them to destroy specific cells, and infusing them back into the body.
After the therapy, Reid “felt like [she] no longer had lupus.” In the two years since the procedure, her lupus has remained in remission without any drugs. Read more about how CAR-T therapy could be used to help people with autoimmune diseases:
newyorkermag.visitlink.me/8sVHNJ
