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 disorders. The study, published in…
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 breast cancer. As reported by…
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 researchers at the Iowa University’s…
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, estimates of the number of…
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 Tecvayli (Johnson & Johnson’s bispecific…
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 Meeting reveals that lean patients…
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 location proximity to major blood…
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. As reported by Drugs.com, this…
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 be a classic example of…
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 genetic disorders using CRISPR-based therapies.…
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 alter how the medical community…
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 I trial for fibrolamellar carcinoma…
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) for lysosomal storage disorders (LSDs).…
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) with compensated cirrhosis—a population with…
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 them resistant to conventional treatments.…
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 is to cause disease. Developed…
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 the B‑cell receptor that fuels…
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 proteins misfold into amyloid fibrils…
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 as a driver of transplant…
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 aggressive form of leukemia. In…
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 with Takeda’s enzyme replacement therapy…
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 early gains. Hunter syndrome is…
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 article in its original format,…
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 campaign with us “Celebrating life’s…
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 type 1 (NF1) who have…
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