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
Learning Her Again: A Mother’s Understanding of Baseline
Accelerated Pathway Opens for GSK’s Pruritus Solution in China’s Rare Liver Disease Population
FDA Grants Priority Review to Enhertu for HER2‑Positive Early Breast Cancer With Residual Disease
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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.
Chronic Lymphocytic Leukemia
FDA Approves Revolutionary Oral Combination Therapy for Chronic Lymphocytic Leukemia, Offering Patients Freedom from Long-Term Treatment
AbbVie has announced a landmark regulatory milestone with the US FDA’s approval of a supplemental new drug application (sNDA) for the combination of Venclexta (venetoclax)
Rare Disease
First Patient Dosed in Phase 3 CLARITY Trial Evaluating Solriamfetol for MDD With Excessive Daytime Sleepiness
As published in Sleep Review Magazine, Axsome Therapeutics has officially begun dosing patients in its Phase 3 CLARITY trial, a study designed to evaluate solriamfetol
Fibrosis
Breaking the Fibrosis Barrier: How Fibrocor Therapeutics Is Revolutionizing Kidney Disease Treatment
Fibrosis, the accumulation of scar tissue in organs, disrupts normal organ function and leads to progressive impairment. Currently, there is no cure, and existing treatments
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On World Kidney Day, we're reminded of the vital role our kidneys play in keeping our bodies healthy- from filtering waste and balancing fluids to support overall wellness. It's also a time to raise awareness about Chronic Kidney Disease, a condition that affects millions of people worldwide, often without noticeable symptoms in its early stages.
Today encourages us to prioritize prevention, early detection, and healthy habits that protect kidney health. Through education, awareness, and compassionate care, we can support those affected and help ensure healthier futures for communities everywhere. 🫘 🌎
#kidneyhealth #PreventKidneyDisease #PatientWorthy
Today we recognized World Alloimmunization and Hemolytic Disease Awareness Day, a time to raise awareness about maternal antibodies and their impact on pregnancy. Conditions like Hemolytic Disease of the Fetus and Newborn (HDFN) can affect babies before they are even born, but with early screening, education, and compassionate care, families can be better supported and protected.
Every expectant parent deserves the knowledge and care that helps give their baby the safest possible start. Awareness, advocacy, and early testing can truly make a life-saving difference. 💜 🤰
#MaternalHealth #prenatalcare #PatientWorthy
Tardive dyskinesia (TD) causes involuntary movements—often affecting the face, mouth, tongue, or limbs—and can develop after long-term use of certain medications. Because symptoms may appear gradually and be mistaken for something else, TD can go unrecognized.
Awareness helps people recognize the signs, start conversations with their healthcare providers, and seek the care and support they need. If you'd like to share your story, click here: bit.ly/4dV7gru
#tardivedyskinesia #TD #shareyourstory #PatientWorthy
