Author Archives: Marisa Wexler MS

Sleep Issues Common, Rarely Addressed, White Paper Asserts

People with Prader-Willi syndrome (PWS) and other diseases commonly experience sleep problems, and the problems are rarely addressed effectively by healthcare professionals, according to a white paper published by Harmony Biosciences. Excessive daytime sleepiness, or EDS, is a common symptom experienced by people with rare diseases…

Differences Due to PWS Genetic Class, Growth Hormone Use Described

Specific genetic subtypes and growth hormone use both influence the physical characteristics, such as head shape and eye appearance, of people with Prader-Willi syndrome (PWS), a large study indicates. The study, “Influence of Molecular Classes and Growth Hormone Treatment on Growth and Dysmorphology in Prader‐Willi Syndrome:…

Soleno Looking to Expand Potential Therapies That Work Like DCCR

Soleno Therapeutics has partnered with Vanderbilt University to develop therapies that work as ATP-dependent potassium channel activators for rare diseases, similar to the diazoxide choline controlled release (DCCR) tablets the company thinks might treat Prader-Willi syndrome (PWS). ATP-dependent potassium (or KATP) channels are proteins present in multiple body tissues.

Tonix Licenses Via-the-nose Treatment Technology From Inserm

Tonix Pharmaceuticals has licensed technology from the French National Institute of Health and Medical Research (Inserm) that it aims to use for treating failure to thrive in Prader-Willi syndrome. The license will allow Tonix to expand its intranasal — administered via the nose — potentiated oxytocin program to…

INV-101 Granted FDA Rare Pediatric Disease Designation

Inversago Pharma‘s INV-101 has been granted rare pediatric disease (RPD) designation by the U.S. Food and Drug Administration for the treatment of Prader-Willi syndrome (PWS). This designation is given to potential medications targeting serious diseases that primarily affect less than 200,000 Americans ages 18 or younger. If the…

FPWR Awards More Than $900,000 to Nine Grants in PWS

The Foundation for Prader-Willi Research (FPWR) is funding nine projects that intend to improve understanding and help find treatments, including gene therapies, for Prader-Willi syndrome (PWS). In total, FPWR awarded a total of $912,251 in the first round of this year’s research awards, according to a press release.