As reported by PharmaBiz, the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion supporting Orphan Drug Designation (ODD) for BioInvent’s BI-1808, an investigational anti-TNFR2…
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…
As reported on World Pharma News, a collaborative research team from the Medical University of Vienna, HUN-REN Research Centre for Natural Sciences, and Eötvös Loránd University has unveiled a promising…
At the 76th annual AASLD Liver Meeting®, Novo Nordisk presented new findings from a post hoc analysis of the ESSENCE phase 3 trial, highlighting the impact of semaglutide 2.4 mg…
Nexiguran ziclumeran (nex-z), an investigational CRISPR-Cas9 gene therapy for transthyretin amyloid cardiomyopathy (ATTR-CM), continues to demonstrate long-term efficacy in early-phase research, even as its phase III trials remain on hold…
Why Rare Diseases Pose a Diagnostic Challenge As discussed in the Orphanet Journal of Rare Disease, a "rare disease" is defined as a condition affecting fewer than 1 in 2,000…
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…
By Thresa Giles, CEO — Hope & Help Center of Central Florida Since HIV was first identified in 1983, there has been a growing, persistent stigma surrounding it. People have…
As reported on MSN, a preliminary study presented at the American Heart Association’s Scientific Sessions 2025 suggests that a non-invasive method for assessing atrial function could help identify stroke risk…
Merck & Co. announced positive results from two Phase 3 studies of its kidney cancer drug Welireg, signaling potential expansion into earlier stages of disease. As reported on BioPharmaDive, in…
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…
We're honored to bring you this video, shared with us by Danny Gereg, that details some of his journey living with bladder cancer. Danny has stayed resilient and positive, finding…
Research into amyotrophic lateral sclerosis (ALS) is gaining momentum. According to Citeline, early-stage clinical activity surged from 28 new trials in 2023 to 60 in 2024, a remarkable increase for…
Cowden Syndrome. A name that sounds almost gentle, until you realize it carries generations of uncertainty. It’s caused by changes in the PTEN gene and can affect the skin, thyroid,…
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…
In a groundbreaking moment for transplant medicine, Massachusetts General Hospital (MGH) has concluded its second successful xenotransplantation of a genetically modified pig kidney into a human patient. Tim Andrews, a…
A collaborative team from Arc Institute, Gladstone Institutes, and UCSF has unveiled a groundbreaking epigenetic editing platform that could revolutionize next-generation cell therapies. Published in Nature Biotechnology on October 21,…
Sanofi has announced encouraging results from a Phase 2 clinical trial of efdoralprin alfa, an investigational therapy for alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder affecting the liver and…
Sanofi has announced encouraging results from its Phase 2 ElevAATe clinical trial evaluating efdoralprin alfa (SAR447537), a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, for the treatment of emphysema caused…
Women living in areas of the United States with poor air quality, particularly neighborhoods with high motor vehicle emissions, face a greater risk of developing breast cancer, according to a…
Denali Therapeutics (Nasdaq: DNLI) announced that the U.S. Food and Drug Administration (FDA) has extended the review period for its Biologics License Application (BLA) for tividenofusp alfa, an investigational therapy…
Genentech, a member of the Roche Group, has unveiled results from two pivotal Phase III trials—MEERKAT and SANDCAT—evaluating the investigational drug vamikibart for uveitic macular edema (UME), a sight-threatening complication…
In a groundbreaking move to reduce the emotional toll and diagnostic delays associated with breast cancer detection, the University of Chicago Medicine has introduced BIOPSY NOW, a patient-centered initiative offering…
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…
In a significant advancement for HIV prevention, the Centers for Disease Control and Prevention (CDC) has issued a strong recommendation for Yeztugo (lenacapavir), Gilead Sciences’ long-acting, twice-yearly pre-exposure prophylaxis (PrEP)…
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