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9 Meters Biopharma acquired global development rights to NM-136, a potential Prader-Willi syndrome (PWS) therapy, from its maker, Lobesity. The therapy candidate, formerly known as LOB-0136, aims to reduce obesity among PWS patients by targeting the glucose-dependent insulinotropic polypeptide (GIP), a molecule that helps sugar enter fat cells.
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
A protein that interacts with insulin growth factor 1 (IGF1) may be important in managing children with Prader–Willi syndrome (PWS) on growth hormone therapy, a study has found. The levels of that protein, called IGFBP7, were found to be higher than normal in PWS children who carry deletions in…
An additional clinical trial into diazoxide choline controlled release (DCCR) tablets as a potential treatment for the insatiable hunger associated with Prader-Willi syndrome (PWS) continues to be seen as necessary by U.S. Food and Drug Administration (FDA) to support a request for DCCR’s approval. But the agency also encouraged Soleno…
The U.S. Food and Drug Administration (FDA) has granted priority review to LV-101 (intranasal carbetocin), an investigational treatment for the distress behaviors and insatiable hunger that accompany Prader-Willi syndrome (PWS). The decision reduces the review period for the medication’s new drug application (NDA) to six months, down from the…
Supervised exercise can improve physical fitness and increase lean body mass in people with Prader-Willi syndrome (PWS), and boost bone health in pediatric patients, according to a review study. These benefits occur even in the absence of weight and fat loss in these patients, who are less active than…
The Foundation for Prader-Willi Research (FPWR) has partnered with My Gene Counsel to safely and responsibly return genetic test results to Prader-Willi syndrome (PWS) patients who have joined the first PWS Genome Project. Launched in May, the PWS genome sequencing project seeks to unveil how variations in a…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
The Foundation for Prader-Willi Research (FPWR) announced its support for ARD-101, an investigational, gut-targeting Prader-Willi syndrome (PWS) therapy, by investing in Aardvark Therapeutics, the treatment’s developer. “FPWR is committed to advancing the development of innovative treatments through multiple channels,” John Walter, CEO of FPWR, said in a…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
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