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Recent changes to a Phase 2 clinical trial of the oral medicine pitolisant to treat excessive daytime sleepiness will make it easier for people with Prader-Willi syndrome (PWS) to participate in the study, according to an announcement from the Foundation for Prader-Willi Research. “In addition to evaluating…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TNX-2900, Tonix Pharmaceuticals’ oxytocin therapy for Prader-Willi syndrome (PWS). Orphan designation is intended to speed the development of potential therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. The status…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…
Tonix Pharmaceuticals has entered a research agreement with Inserm Transfert, the private subsidiary of the French National Institute of Health and Medical Research (Inserm), to study the mechanisms behind the reported benefits of the oxytocin hormone in a mouse model of Prader-Willi syndrome (PWS). Inserm Transfert signed the deal…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…
Dietary supplementation with conjugated linoleic acid led to weight loss in a mouse model of Prader-Willi syndrome (PWS), according to a recently published study. Results were detailed in the journal Nutrients, in the article “Dietary Conjugated Linoleic Acid Reduces Body Weight and Fat in Snord116m+/p−…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
A case of acute kidney injury in a woman with Prader-Willi syndrome (PWS) and intellectual disability was attributed in a recent report to the use of multiple medications. The case shed lights on how vulnerable people with an intellectual disability are to being over-medicated, with the complications…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
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