Prader-Willi Syndrome (PWS) is a complex, rare genetic disorder known for its hallmark features: insatiable hunger, intellectual and behavioral challenges, and a host of physical health concerns. For families and…
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…
Prader-Willi syndrome is a leading cause of obesity in children. The rare disease was discovered over seventy years ago, yet this is the first therapy to be approved by the…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Prader-Willi syndrome (PWS) is a rare genetic disorder with a number of physical, emotional, mental, and behavioral manifestations. Individuals with PWS may exhibit cognitive impairment, hypotonia (poor muscle tone), and…
Written by Maria Picone, TREND Community Ten years ago, my daughter was born with a rare, complex disease known as Prader-Willi syndrome (PWS). PWS is best known for causing insatiable…
The month of May is recognized as Prader-Willi Syndrome (PWS) Awareness Month, and May 27th is International PWS Day. This is a time to spread awareness about this rare disease…
Dr. Seth Lederman founded Tonix Pharmaceuticals with a deep desire to change the treatment landscape for patients. The company’s rich pipeline features therapeutic options for fibromyalgia, post-traumatic stress disorder (PTSD),…
According to a story from prnewswire.com, the pharma company Harmony Biosciences Holdings, Inc., has recently announced the release of data from its phase 2 clinical trial. This trial is evaluating…
Saniona has just announced that their Phase 2b clinical trial examining Tesomet as a treatment for Prader-Willi syndrome (PWS) will be beginning soon. This treatment is a combination therapy which…
Every year, there are numerous unique ways to get involved in the rare disease community and help raise awareness. One example? The Harvest Hope for PWS event -- a virtual…
Hyperphagia, or an abnormally stronger hunger or desire to eat, is one of the characteristics of Prader-Willi syndrome (PWS), a rare genetic disorder. Because of this, patients with PWS often…
There are many reasons why medical research is important. It helps to amplify the patient voice, improve disease-related knowledge, and determine potential treatment goals. Additionally, medical research provides insight into…
Vanderbilt University has just announced a new collaborative agreement with Soleno Therapeutics in aim to develop KATP channel activators as a therapeutic for rare diseases. This therapy has already been…
Tonix Pharmaceuticals and Inserm, which is the French National Institute of Health and Medical Research, have entered into a licensing agreement in which Tonix will hold the license to oxytocin-based…
According to a story from GlobeNewswire, the biopharmaceutical company Soleno Therapeutics, Inc. recently hosted a Key Opinion Leader (KOL) webinar, which was focused primarily on the latest updates to its…
Soleno Therapeutics has announced that they will be hosting a Key Opinion Leader (KOL) webinar on Thursday, February 4 at noon EST. This webinar will focus on Prader-Willi syndrome, specifically…
According to a story from BioNews, a recent study has revealed more information about the maternal protein SMCHD1, and impacts to the normal function of this protein could play a…
Just around a month ago, the National Organization for Rare Disorders (NORD) offered a webinar on how to create a patient registry or start a nonprofit organization. NORD's webinar, part…
Within the last week, biotechnology company Levo Therapeutics announced results from the Phase 3 CARE-PWS clinical trial. During the trial, researchers examined the efficacy, safety, and tolerability of LV-101…
In an interview with Healio, Dr. Jennifer Miller says that there are signs of hope for improved outcomes in patients living with Prader-Willi syndrome, a rare genetic disorder. Dr. Miller…
According to a story from Malvern Daily Record, the biopharmaceutical company Millendo Therapeutics, Inc. recently released data from its phase 2b clinical trial. This study was testing the company's experimental…
According to a story from labblog.uofmhealth.org, one of the defining features of Prader-Willi syndrome, a rare disorder, is hyperphagia, which is extreme overeating and obsession with food. While this is…
Hyperphagia is an extreme hunger and obsession with food, and it is a symptom of Prader-Willi syndrome. While many of the other effects of this condition have treatments, this…
Although even a regular video of twin baby girls snuggling can be engaging and cute, there are two twin girls in the UK whose bond will warn your heart.…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
