In the remote town of Serrinha dos Pintos in northeastern Brazil, a rare genetic disorder once shrouded in mystery has shaped the lives of many families for generations. As reported…
Since its inception in 2015, PatientWorthy.com has emerged as a beacon of hope, information, and support for the global rare disease community. From humble beginnings with its very first post,…
An article titled “Accelerated Approval as the New Norm in Gene Therapy for Rare Diseases” from DIA Global Forum digital magazine’s May 2025 issue explores the evolving landscape of regulatory…
Soleno Therapeutics recently announced new findings regarding its investigational drug VYKAT™ XR (diazoxide choline extended-release tablets) for the treatment of Prader-Willi syndrome (PWS), as detailed at GlobeNewswire. The data, which…
Right now, more people than ever are hoping for a kidney transplant — but the system is struggling to keep up. According to Becker’s Clinical Leadership, even a small increase…
According to the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), eating the right diet is essential to managing chronic kidney disease. The NIDDK recommends that patients find…
In honor of Kidney Awareness Month, we would like to congratulate the National Kidney Foundation on celebrating 75 years as an organization fighting against kidney disease and for kidney disease…
Measles cases in Texas and New Mexico have doubled according to Becker's Clinical Leadership surpassing 200. 30 cases have been confirmed in New Mexico, mostly in Lea County which is…
The U.S. Food and Drug Administration (FDA) has approved GSK's combination meningococcal vaccine, Penmenvy, for individuals aged 10 to 25 as reported by Biopharma Dive. This vaccine offers protection against…
Heal Canada is a non-profit dedicated to “patient advocacy, education and collaboration among patient advocacy groups and other healthcare stakeholders.” As part of their extensive offerings of support for patients…
Novo Nordisk plans to seek approval in the US and the EU this year for their drug Mim8 for use on pediatric patients with Hemophilia A after promising results from…
Spinogenix has reported that its Phase II trial of SPG601 for Fragile X Syndrome (FSX) has met its primary goal, according to Clinical Trials Arena. FXS is a genetic disorder…
As reported by Clinical Trials Arena, Genentech and its subsidiary Roche, provided positive Phase III results with the Food and Drug Administration, or the FDA, of their REGENCY trial (NCT04221477)…
In this insightful article, Sierra Domb, Founder and CEO of the Visual Snow Initiative (VSI), a nonprofit organization dedicated to raising global awareness, providing education and resources, advocating for patients,…
According to World Pharma News, an AI technique called machine learning was able to identify a lifesaving treatment for a patient diagnosed with idiopathic multicentric Castleman’s disease (iMCD) by scanning…
Researchers from the National Institutes of Health and the Human Hereditary and Health in Africa (H3Africa) Kidney Research Network reported that people from West Africa with one genetic risk variant…
Living with a chronic condition is never easy, but living with two can feel like an insurmountable challenge. My journey with epilepsy and endometriosis has been a test of resilience,…
Athletes experiencing multiple brain injuries now have more to worry about than ever. Excessive traumas to the brain common in sports like boxing and football, are now being linked to…
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