News

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…

The prevalence of gastro-esophageal reflux disease (GORD), a condition also known as GERD, was found to be high among patients with Prader-Willi syndrome (PWS), a new study shows. Researchers also found a strong link between tooth wear, or loss of tooth substance, and the prevalence of GORD, as well…

Insys Therapeutics’ Phase 2 trial to evaluate the safety and effectiveness of its cannabidiol (CBD) oral solution in pediatric patients with Prader-Willi syndrome (PWS) will soon begin recruiting participants. More information about the study and dates of enrollment will be announced on the clinical trial’s official webpage, found here (NCT02844933).

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

Saniona obtained regulatory approval to initiate the second part of a Phase 2a study evaluating the investigative therapy Tesomet (tesofensine/metoprolol) in adolescents with Prader-Willi syndrome (PWS) after positive results from the first part of the study with adult patients. Prader-Willi is a genetic disorder that begins in childhood. People…

Patients with Prader-Willi syndrome (PWS), diabetes, and fat accumulation in the pancreas may benefit from a combined treatment of two therapies that treat diabetes: tofogliflozin (which has not yet been approved in the U.S.) and dulaglutide, according to a Japanese case report. The study, “…