In a statement by the FDA, the U.S. Food and Drug Administration has authorized Waskyra (etuvetidigene autotemcel), the first gene therapy designed to treat Wiskott-Aldrich
The biotech sector witnessed significant momentum as four clinical-stage companies announced compelling data across muscular dystrophy, immunology, and obesity treatment areas, triggering substantial stock market
As reported by MedicalXpress, Researchers have uncovered a surprising immune function in specialized intestinal cells that may reshape our understanding of gut health and autoimmune
Genentech announced landmark clinical trial results demonstrating that giredestrant, an investigational selective estrogen receptor degrader (SERD), significantly outperforms standard endocrine therapy in treating early-stage ER-positive
Madrid, December 3, 2025 – The research laboratory of the Dravet Syndrome Foundation was one of the initiatives awarded 3,000 Euros in the 11th edition
A rare and life-threatening kidney disease affecting young adults and children finally has an effective and approved therapy due to the pioneering and leadership of
A team of doctors at NYU Langone transplanted a pig kidney into a brain-dead patient and reversed its rejection twice during the 61-day study. Currently,
As reported on BioPharmaDive, new clinical trial data presented at the American Society of Hematology (ASH) annual meeting suggests that an early treatment regimen combining
Key Insight: As reported by Healio, metabolic dysfunction-associated steatotic liver disease (MASLD) is not exclusive to individuals with obesity. New research presented at The Liver
There may be several reasons that HCC cancer of the liver cannot be removed. It may be due to the size of the tumor, or
Parabilis Medicines has achieved a significant milestone in cancer drug development, receiving FDA Fast Track designation for FOG-001, a groundbreaking first-in-class therapy targeting desmoid tumors.
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
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
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
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
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)
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)
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
Getting a diagnosis can feel overwhelming, especially when the process moves quickly. Understanding why certain tests are done and how treatment decisions are made can help bring a little clarity to a very uncertain moment.
This post walks through the big picture — but everyone’s path with DLBCL is personal. Questions are valid, second opinions are okay, and support matters at every step! If you're interested in sharing your story with DLBCL, click here: bit.ly/4dV7gru
#DLBCL #Lymphoma #ShareYourStory #PatientWorthy
Today we recognize Paget’s Disease Awareness Day, shining a light on a rare and often misunderstood condition. Increased awareness leads to earlier diagnosis, better care, and stronger support for those affected. #PagetsDisease #PatientWorthy #RareDisease
#ShareYourStory here: bit.ly/4dV7gru
We're honored to share this story from our friends at Elephants & Tea, written by Michelle and detailing her experiences with Diffuse Large B-Cell Lymphoma. Read here:
patientworthy.com/2026/01/11/it-was-all-a-dream/
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