Rare Disease
The Ever growing Field of Molecular Glues
Breaking a system into small independent and interchangeable modules that can perform certain functions is called modularity. For many years the KRAS oncogene proved to
Repurposing Breast Cancer Drug Offers New Hope for Acute Myeloid Leukemia Patients
- January 6, 2026
Living with gMG: Lessons from William, a Nurse and Patient
FDA Approve Drug That Targets Root Cause of Life-Threatening Kidney Disease Affecting Children and Young Adults
Tonsillectomy Plus Immunosuppression Improves Outcomes in High-Risk IgA Nephropathy Among Caucasians
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Gene Therapies
FDA Launches New Trial Protocol for Customized Gene Therapies
As reported on MedicalXpress, esearchers at the Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania are pioneering a new approach to treating rare
Parkinson's Disease
Revolutionizing Parkinson’s Care: A New Immunological Approach Delivers Striking Clinical Evidence
In a significant development for neurodegenerative disease treatment, BioPharma Dive reports that AC Immune has unveiled compelling evidence that its novel immunological strategy may fundamentally
fibrolamellar carcinoma (FLC)
Universal-Target Vaccine Shows Early Promise in Rare Liver Cancer Affecting Youth
An experimental cancer vaccine from the Johns Hopkins Kimmel Cancer Center and the Bloomberg~Kimmel Institute for Cancer Immunotherapy has delivered encouraging results in a phase
Rare Disease
Chiesi Group Partners with Aliada Therapeutics to Advance Blood-Brain Barrier Technology for Rare Diseases
Chiesi Group has entered an exclusive licensing agreement with Aliada Therapeutics, a subsidiary of AbbVie, to develop therapies that can cross the blood-brain barrier (BBB)
Metabolic dysfunction-associated steatohepatitis
Rezdiffra (Resmetirom) Shows Sustained Benefits in MASH Cirrhosis Over Two Years
As reported on Healio, new findings presented at The Liver Meeting highlight the potential of resmetirom (Rezdiffra, Madrigal Pharmaceuticals) in managing metabolic dysfunction-associated steatohepatitis (MASH)
Diffuse Large B-cell Lymphoma
Sugar-Coated Deception: Discovery of Aggressive Lymphoma Subtype Opens Door to Personalized Cancer Treatment
Researchers at the University of Southampton have uncovered a hidden culprit in blood cancer, a unique sugar molecule that disguises aggressive lymphoma cells and renders
Rare Disease
PopEVE Aims to Fast-Track Rare Disease Diagnoses by Ranking Genetic Variant Severity
A new artificial intelligence model, popEVE, could accelerate diagnoses for rare, single-variant genetic diseases by scoring how likely each genetic change in a patient’s genome
Diffuse Large B-cell Lymphoma
Mann-type DLBCL: Sugar Signature Reveals High-Risk Lymphoma and New Therapeutic Avenues
University of Southampton researchers have identified a distinct subtype of diffuse large B‑cell lymphoma (DLBCL), termed “Mann-type DLBCL,” marked by a unique mannose sugar on
Light chain disease
The FDA Grants Traditional Approval for Daratumumab Regime in Amyloidosis
Light chain amyloidosis (AL) is a progressive and rare disease whereby abnormal plasma cells found in the blood and urine over-produce light chain proteins. These
Rare Disease
Houston Methodist Researchers Identify T Cells That Attack Transplanted Organs
The findings were recently published in Nature Immunology and reported by Precision Medicine suggesting that a factor called Interferon Regulatory Factor 4 (IRF4) takes responsibility
Leukemia
RFK’s Granddaughter, Tatiana Schlossberg, Reveals Diagnosis of Rare Blood Cancer
As reported on NPR, Tatiana Schlossberg, journalist and granddaughter of former U.S. President John F. Kennedy, has disclosed that she is battling a rare and
Thrombotic Thrombyctopenic Purpura
FDA Investigates Death Linked to Takeda’s Adzynma Therapy
As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has launched an investigation into reports of serious adverse events, including one fatality, associated
MPS II (Hunter Syndrome)
First-in-Human Gene Therapy Offers Hope for Hunter Syndrome: Oliver’s Remarkable Turnaround
Three-year-old Oliver Chu has stunned clinicians after becoming the first person with Hunter syndrome (MPS II) to receive an experimental gene therapy and showing striking
Brain Cancer
“The Part That Died”
Editor’s Note: Patient Worthy is pleased to share this article from our friends at Elephants & Tea, originally written by Cecily Liu. To see the
Leukodystrophy
Marc Nolan’s Announces Partnership with the United Leukodystrophy Foundation
The United Leukodystrophy Foundation (ULF) is proud and excited to announce a partnership with Marc Nolan, a Chicago-based footwear brand, who is launching a new
Neurofibromatosis Type 1
FDA Approves Koselugo for Adults with Neurofibromatosis Type 1
The U.S. Food and Drug Administration (FDA) has approved Koselugo (selumetinib), an oral MEK inhibitor developed by Alexion, AstraZeneca Rare Disease, for adults with neurofibromatosis
Diffuse Large B-cell Lymphoma
NHS Fast-Tracks Glofitamab, Expanding Curative Options for Aggressive Lymphoma
Hundreds more patients in England with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) will gain earlier access to glofitamab (Columvi) after new guidance from
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DLBCL is an aggressive blood cancer where B-cells grow uncontrollably and spread rapidly through lymph tissue. Swipe to understand how it develops and why early detection matters, and visit patientworthy.com for more information!
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Repurposing Breast Cancer Drug Offers New Hope for Acute Myeloid Leukemia Patients bit.ly/3LkNcVJ Read now at PatientWorthy.com
#PatientWorthy #AcuteMyeloidLeukemia #AML
