ADHD
Landmark Review Identifies Most Effective Evidence‑Based Treatments for ADHD
As reported by Technology, a major evidence synthesis published in The BMJ has delivered the clearest picture to date of which therapies offer the strongest
FDA Places Clinical Holds on REGENXBIO’s MPS Gene Therapy Programs After Tumor Detected in Trial Participant
- February 6, 2026
James’ Story: Navigating Life with Myelofibrosis
Roche’s Obesity Drug Shows Promise Against Lilly and Novo Nordisk
EMA Endorses First Immunotherapy Option for Advanced Anal Cancer
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Rare Disease
Pediatric Palliative Care
Editor’s Note: Patient Worthy is honored to bring you this article, originally written by Kelsey Stanczyk, and shared with us by our friends at the
IgA Nephropathy (IgAN)
New Drug Targets Root Cause of IgAN by Blocking Immune Cell Signals
IgA nephropathy represents one of the most pressing kidney health challenges facing young adults worldwide. This progressive kidney disease develops when the immune system malfunctions,
non-small cell lung cancer
AstraZeneca’s LATIFY Trial Misses Primary Endpoint in Previously Treated Advanced NSCLC
As reported on PharmaBiz, AstraZeneca’s phase III LATIFY trial has fallen short of its primary objective, with the combination of the ATR inhibitor ceralasertib and
Canavan disease
Oligodendrocyte‑Targeted Gene Therapy Shows Breakthrough Potential in Canavan Disease
As reported on InsidePrecisionMedicine, a new gene therapy aimed directly at the cells responsible for myelination is showing unprecedented promise for children with Canavan disease,
Prostate Cancer
Hidden Protein Markers Offer New Hope for Predicting Prostate Cancer Across Diverse Communities
Prostate cancer doesn’t affect all men equally. Medical researchers have long known that men with African ancestry experience significantly higher rates and more severe outcomes
Chronic Inflammatory Demyelinating Polyneuropathy
MHRA Grants New Indication for argenx’s Vyvgart in Chronic Inflammatory Demyelinating Polyneuropathy
As reported by The Pharma Letter, the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted a new indication for argenx’s Vyvgart (efgartigimod alfa),
Rare Disease
Why Palliative Care Shouldn’t Wait: A Nurse and Mother’s Story
Editor’s Note: Patient Worthy is proud to share this story from the Courageous Parents Network, originally written by Kelsey Stanczyk. When I first became a
ADHD
Rethinking ADHD: How Brain Scans Reveal Stimulants Work Through Motivation, Not Focus
For decades, doctors have prescribed stimulant medications like Ritalin and Adderall to millions of children with ADHD, operating under the assumption that these drugs directly
Cystic Fibrosis
Nick and Rene’s Caregiver Story for Their Daughter with Cystic Fibrosis
I’ve learned something brutal over the last few weeks: Most people cannot tolerate the reality of a child nearly dying. So they dilute it. They
HIV/AIDS
From HIV Treatment to Rare Genetic Disease: Nucleoside Therapy Enters a New Era
As reported on Forbes, a class of drugs first introduced to combat HIV is now demonstrating unexpected promise for patients with rare genetic and mitochondrial
Psoriasis
Once-Daily Zasocitinib Delivers Clear Skin for Psoriasis Patients: Takeda’s Landmark Phase 3 Victory
Takeda Pharmaceutical Company has announced breakthrough results from Phase 3 trials of zasocitinib (TAK-279), an innovative oral treatment for moderate-to-severe plaque psoriasis that offers patients
Rare Disease
Biohaven’s BHV‑7000 Falls Short in Midstage Depression Trial, Refocusing Pipeline Strategy
As reported on FierceBioTech, Biohaven closed out the holiday period with disappointing news: its investigational Kv7.2/7.3 potassium‑channel modulator, BHV‑7000, did not demonstrate efficacy in a
Non-Hodgkin's Lymphoma
“The Aftermath: Nobody Told Me This Part Would Hurt Too: – Megan’s Fight Against Non-Hodgkin’s Lymphoma
Editor’s Note: Patient Worthy is proud to share this story from our friends at Elephants & Tea. To see the story in its original format,
Insomnia
Breaking New Ground: Daridorexant Offers Hope for Menopausal Insomnia
Menopause-related insomnia affects approximately one in four women during the menopausal transition, yet it remains significantly underdiagnosed and undertreated. A new analysis published in Maturitas
Hunter Syndrome
UNC Researchers Report Promising Advances Toward First Brain‑Targeting Enzyme Therapy for Hunter Syndrome
As reported by NewsWise, new clinical study led by investigators at the University of North Carolina at Chapel Hill is offering rare hope for families
Alzheimer's disease
Fast-Track FDA Review Accelerates Path to First Treatment for Alzheimer’s-Related Agitation
Axsome Therapeutics has reached a significant regulatory milestone with the US Food and Drug Administration’s acceptance of a supplemental New Drug Application (NDA) for AXS-05
Rare Disease
FDA Approves First At-Home Brain Stimulation Device for Depression
As reported by Forbes, the U.S. Food and Drug Administration has granted approval to Flow Neuroscience for the first prescription brain stimulation device designed for
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Feeding Tube Awareness Week is about visibility, understanding, and respect. Feeding tubes are not a last resort or a failure—they’re a medical tool that provides nourishment, strength, and life. Behind every tube is a person deserving of dignity, patience, and compassion. #FeedingTubeAwarenessWeek #ShareYourStory #PatientWorthy
Meet Amber! She was diagnosed with Granulomatosis with Polyangiitis (GPA) in 2017. Through traditional medicine, holistic care, and fierce self-advocacy, Amber is now in remission! She shares her story to educate medical professionals and empower others living with GPA. For Rare Disease Month, her message is clear: we are not alone. Together we share our stories and learn from each other, and knowledge is power! Thanks Honey I Roam for sharing your story with us!
#granulomatosiswithpolyangitis #Wegeners #rarediseasemonth #RareDisease #rarebutnotalone #WeCareAboutRare #SHAREYOURSTRIPES #shareyourstory #PatientWorthy
Mississippi Metabolics Foundation #RareDiseaseThis is a monumental day for rare disease patients!!! All our hard work has been realized.
