Brain Cancer
Learning Her Again: A Mother’s Understanding of Baseline
Editor’s Note: Patient Worthy is honored to share this caregiver story with you, originally written by Aisling Melton, Parent Champion at Courageous Parents Network. In
Heather and Nathan’s Journey with Huntington’s Disease
My Journey with Ehlers-Danlos Syndrome
Faye’s Journey with Amyloidosis
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Primary Biliary Cholangitis
Accelerated Pathway Opens for GSK’s Pruritus Solution in China’s Rare Liver Disease Population
GlaxoSmithKline has achieved a significant regulatory milestone as China’s National Medical Products Administration granted priority review status to linerixibat, an investigational treatment for cholestatic pruritus
Breast Cancer
FDA Grants Priority Review to Enhertu for HER2‑Positive Early Breast Cancer With Residual Disease
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has accepted and granted Priority Review to AstraZeneca and Daiichi Sankyo’s supplemental Biologics License
Amyloidosis
Two Intellia Clinical Trials Released by the FDA
Two of Intellia Therapeutics’ clinical trials that had been on hold since October 2025 have now been released by the FDA. According to The New
Hunter Syndrome
Gene Therapy Setback: REGENXBIO’s RGX-121 Faces Regulatory Hurdles in Ultra-Rare Disease Treatment
REGENXBIO encountered a significant regulatory obstacle when the FDA issued a complete response letter regarding its biologics license application for RGX-121, an investigational gene therapy
Parkinson's Disease
Serina Therapeutics Begins First-in-Patient Dosing in Phase 1b Trial of SER‑252 for Advanced Parkinson’s Disease
As reported on RTT News, Serina Therapeutics has initiated patient dosing in its Phase 1b registrational study of SER‑252, marking a significant milestone for the
Phenylketonuria
New Gene Editing Program Aims to Address Root Cause of PKU
For families affected by phenylketonuria (PKU), newborn screening can mean the difference between a healthy future and lifelong complications. PKU is a rare inherited metabolic
Tardive Dyskinesia
From Psychiatric Bind to Clinical Solution: The VMAT2 Revolution in Treating Tardive Dyskinesia
Tardive dyskinesia represents one of medicine’s cruelest ironies: a side effect born from the very medications designed to restore psychiatric health. For generations, treating this
Brain Cancer
Brain Cancers: FDA Approves Pembrolizumab Combo
Astrocytoma and glioblastoma are fast-growing brain cancers that often return after surgery. Survival for astrocytoma patients is generally four to five months. Medications that activate
Bipolar disorder
Breaking New Ground: Milsaperidone Offers Patients a Fresh Option for Serious Mental Illness
Vanda Pharmaceuticals has achieved a significant regulatory milestone with the FDA’s approval of milsaperidone (Bysanti), a newly designated atypical antipsychotic medication poised to help adults
Rare Disease
FDA Escalates Scrutiny of GLP‑1 Compounders as Industry Reports Key Clinical and Regulatory Updates
As reported by BioPharmaDive, he Food and Drug Administration (FDA) has intensified its oversight of companies marketing compounded GLP‑1 therapies, while Intellia Therapeutics, United Therapeutics,
Lynch syndrome
A New Shield Against Cancer: The Lynch Syndrome Vaccine
For people living with Lynch syndrome, the threat of cancer is a constant shadow. This inherited condition affects about one in 300 people, making them
Rare Disease
Blood-Based circRNA Marker Shows Promise for Predicting Antidepressant Response
As reported on MedicalXpress, a new study published in Molecular Psychiatry suggests that a specific circular RNA found in blood, known as CDR1as, may help
Prader-Willi Syndrome
Aardvark Pauses Late‑Stage Prader–Willi Trial After Cardiac Signals Emerge
As reported on BioPharmaDive, Aardvark Therapeutics has halted dosing and enrollment in its late‑stage HERO trial for ARD‑101, a first‑in‑class treatment candidate for Prader–Willi syndrome
Breast Cancer
Myth or Truth? Avoid Soy If You Have a Hormone-Positive Cancer
Editor’s Note: Patient Worthy is pleased to bring you this article, originally shared with us by our friend Julie Lanford, The Cancer Dietitian. To see
Breast Cancer
Breaking New Ground: FDA Green-Lights Novel Breast Cancer Treatment Targeting Treatment-Resistant Tumors
The landscape of advanced breast cancer treatment is shifting. Genentech’s filing acceptance of giredestrant, an experimental oral medication, represents a pivotal moment for thousands of
Huntington's disease
Vico Therapeutics Begins Twice‑Yearly Dosing of VO659 in Phase 1/2 Trial for Huntington’s Disease and Spinocerebellar Ataxias
As reported in Business Wire, Vico Therapeutics has initiated patient dosing in an expanded cohort of its Phase 1/2a clinical study evaluating VO659, an antisense
Myelofibrosis
James’ Story: Navigating Life With Myelofibrosis
Editor’s Note: This article was shared with us by our friends at Heal Canada. To see the article in its original format, please click here.
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We're honored to share Heather's story as a caregiver for her husband Nathan, who is living with Huntington's disease. Read here:
patientworthy.com/2026/03/28/heather-and-nathans-journey-with-huntingtons-disease/
#PatientWorthy #shareyourstory #patientstory #HD #huntingtons #HuntingtonsDisease
Colorectal Cancer Awareness Month: Colorectal cancer is preventable, treatable, and survivable—especially when caught early. Swipe through these 5 important facts about prevention, screening, and advances in treatment. #ColorectalCancerAwarenessMonth #colorectalcancer #FactFriday #PatientWorthy
#shareyourstory with us here: bit.ly/4dV7gru
Understanding Testing for Non-Small Cell Lung Cancer with HER2 Mutation- ✨
When diagnosed with NSCLC, testing for genetic mutations like HER2 is key too choosing the right treatment. From biopsies to advanced tests like NGS & FISH, identifying the HER2 mutation helps doctors target therapies that can make a difference. #LungCancerAwareness #HER2Mutation #PatientWorthy
