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 up to 80% more likely…
As reported on BioSpace, Moderna has entered a commercialization agreement with Recordati valued at up to $160 million to support late‑stage development and future market introduction of mRNA‑3927, an investigational…
As reported on Science Daily, a breast cancer vaccine tested more than 20 years ago is drawing renewed scientific interest after researchers discovered that every participant in the original trial…
As reported on Healio, glucagon-like peptide-1 (GLP-1)–based therapies are rapidly reshaping the landscape of diabetes and chronic kidney disease (CKD) management. Originally developed for glycemic control, these agents now play…
Immunoglobulin A nephropathy (IgAN) remains one of the most common primary glomerular diseases worldwide, yet effective treatments are limited. A new study, recently published in Frontiers, offers fresh evidence that…
Brussels — On February 24, 2026, the acclaimed Netflix documentary The Remarkable Life of Ibelin received the Media & Awareness Raising Award at the EURORDIS Black Pearl Awards. The honor…
As reported on PM Live, a new clinical trial hub focused on epidermolysis bullosa (EB) has been established at University Medical Center Groningen (UMCG) in the Netherlands through a collaboration…
To the patient, the parent, the caregiver, and the person in the "limbo" of waiting: I am one of you. My name is Aaron Jackson, and this is my story.…
As reported on MedPage Today, an oral targeted therapy may offer a safe and effective alternative to injectable treatment for children with achondroplasia, according to new phase II data published…
As reported by NewsWise, new clinical study led by investigators at the University of North Carolina at Chapel Hill is offering rare hope for families affected by Hunter syndrome, a…
CRISPR Therapeutics has announced encouraging early results for zugocaptagene geleucel (zugo-cel), its investigational allogeneic CAR T therapy targeting CD19, in both autoimmune diseases and hematologic malignancies. Autoimmune Disease Progress In…
As reported on PharmaBiz, Amneal Pharmaceuticals, in collaboration with mAbxience, has received U.S. Food and Drug Administration (FDA) approval for two denosumab biosimilars: Boncresa (referencing Prolia) and Oziltus (referencing Xgeva).…
As reported on PharmaBiz, GENESIS Pharma has announced an exclusive agreement with Otsuka Pharmaceutical Europe Ltd. (OPEL) to distribute and commercialize donidalorsen across 14 Central and Eastern European countries, including…
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 syndrome (WAS), a rare and…
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).…
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…
I was introduced to Amber Olsen, the “mama bear” in my new documentary film, The Zebra and the Bear, in the spring of 2016. A mutual friend asked if I…
As reported on PharmaBiz, the National Organization for Rare Disorders (NORD) has announced a new request for proposals (RFP) aimed at nonprofit patient advocacy groups interested in launching rare disease…
Prader-Willi syndrome (PWS) is a rare genetic disorder affecting approximately 1 in 10,000–30,000 individuals worldwide. It is the leading syndromic cause of obesity, with an estimated 400,000 cases globally and…
A groundbreaking discovery by an international team led by Shinghua Ding at the University of Missouri has revealed a previously unknown genetic disorder affecting motor neurons and muscle control. As…
A Wake Forest University School of Medicine study reports that two existing medications (empagliflozin (Jardiance), a diabetes drug, and intranasal insulin) could improve brain health in individuals with mild cognitive…
Author's Note: Patient Worthy began following Natera following their presentation at the American College of Medical Genetics conference many years ago. Natera has been known for its advances in prenatal…
As reported by drugs.com, at the American College of Rheumatology (ACR) Convergence congress, Novartis presented encouraging results from its Phase III NEPTUNUS-1 and NEPTUNUS-2 trials, highlighting the potential of ianalumab…
A groundbreaking study from the MRC London Institute of Medical Sciences (LMS) is reshaping how researchers approach treatment development for rare genetic disorders. Led by Dr. André Brown and the…
Editor's Note: Patient Worthy is proud to share this story from GACI Global, originally published by the Italian Osservatorio Malattie Rare (OMaR). ARHR2 – Autosomal Recessive Hypophosphatemic Rickets Type…
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