Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose ... Read more
Updates
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Nonprofit Group Works to Raise Rare Disease Awareness in India
With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it ... Read more -
At-Home Dialysis Can Manage Kidney Disease in Prader-Willi Patients, Case Report Shows
Peritoneal dialysis (PD), a blood filtration procedure that can be done at home, has been used successfully over a long period to manage kidney ... Read more -
Study Outlines Rare Case of PWS Patient with Ear Canal and Bone Infection
A new study described a rare case of a young man with Prader-Willi syndrome (PWS) with an invasive infection of the ear canal and ... Read more -
Report Details Use of Denosumab to Treat Osteoporosis in PWS Patient
A case report suggested that treatment with denosumab (commercial names: Prolia, Xgeva) could be effective for patients with Prader-Willi syndrome (PWS), osteoporosis, and a history of ... Read more
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UConn, Foundation for Prader-Willi Research Create Stem Cell Biobank
UConn Health and the Foundation for Prader-Willi Research (FPWR) announced the creation of a ... -
New Blood Test May Help Diagnose Prader-Willi Syndrome, Other Complex Disorders
A new blood test called EpiSign may help identify complex and hard-to-diagnose genomic imprinting disorders such as Prader-Willi ... -
Cactus Extract Reduces Appetite in PWS Mouse Model, Study Reports
Treatment with an Indian cactus extract reduces appetite in a mouse model of Prader-Willi ... -
Ghrelin Receptor Agonist May Improve Survival of Infants with PWS, Mouse Study Suggests
A compound that could mimic ghrelin hormone activity and trigger its receptor signals could ... -
Millendo Starts Phase 2b/3 Study Evaluating Livoletide as PWS Treatment
Millendo Therapeutics has launched a Phase 2b/3 clinical trial investigating the safety and efficacy ... -
Board Recommends Continuation of Phase 3 Trial of Soleno’s DCCR Treatment for PWS
The Data Safety Monitoring Board recommended the continuation of Soleno Therapeutics’ Phase 3 clinical ...
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Lean Muscle Mass Factors Strongly in Bone Health, Study Using Prader Willi Patients as Model Finds
News
Lean muscle mass is a strong predictor of bone mineral density, or bone health, research that used Prader-Willi syndrome (PWS) as a genetic model to determine which factors influence such density reports. In fact, lean mass was found to ... Read more