News

As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…

Biotech company Saniona recently announced that the Phase 1 pharmacokinetic study of a new tablet therapy candidate for Prader-Willi syndrome, called Tesomet (tesofensine/metoprolol), produced promising results in healthy men. Prader-Willi syndrome (PWS) is an inherited disorder that creates feelings of constant hunger, which often leads to obesity and type 2 diabetes.

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

Perinatal complications, including hypotonia (low muscle tone) and feeding difficulties, are clinical features more common among newborns with Prader-Willi syndrome, compared with the general population, according to a new study. The study with that finding, “Multicentre study of maternal and neonatal outcomes in individuals with Prader-Willi syndrome,”…

Motor disability is a common problem in patients with Prader-Willi syndrome (PWS) and may negatively impact their caregivers’ quality of life, particularly physical and emotional problems, researchers from Taiwan reported. Their study, “Motor performance in Prader-Willi syndrome patients and its potential influence on caregiver’s…

A multicenter, Phase 3 clinical trial assessing diazoxide choline controlled-release (DCCR) for the treatment of Prader-Willi syndrome (PWS) recently was initiated, the trial’s sponsor, biopharmaceutical company Soleno Therapeutics, announced. Soleno is currently developing DCCR as a once-daily oral tablet for the treatment of PWS. The tablet is made of a…

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

A 24-week exercise program showed encouraging results in controlling obesity and diabetes in a young man with Prader-Willi syndrome, according to a case study. The intensive exercises resulted in reduced body weight, fat mass, and mean blood glucose. And an increase in blood insulin levels allowed researchers to stop…

Zafgen‘s investigative therapy ZGN-1258, under development as a treatment for Prader-Willi syndrome, is being evaluated in preclinical studies as it moves toward clinical trials in humans, according to the biopharmaceutical company. The results of this initial phase are expected to support the submission of an investigational new drug…