News

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

Children with Prader-Willi syndrome (PWS) as young as 4 are now eligible to participate in the ongoing Phase 3 trial testing diazoxide choline controlled-release (DCCR) tablets for PWS initiated by Soleno Therapeutics earlier this year. The original minimum age limit for enrollment was 8. Feedback from the U.S. Food…

Soleno Therapeutics announced the awarding of a patent for diazoxide choline controlled-release (DCCR) tablets, the company’s investigational therapy for Prader-Willi syndrome (PWS) being evaluated in a new Phase 3 trial. The patent covers “pharmaceutical formulations of diazoxide and diazoxide choline” to treat patients with poor lean body mass and a lean…

A variation in the growth hormone receptor (GHR) gene, specifically the lack of a coding sequence called exon 3, does not seem to increase the risk for scoliosis in Prader-Willi syndrome (PWS) patients, a small study reports. Exon 3 in the GHR gene was previously shown to be associated with increased growth…

A recent study from Australia revealed that patients with Prader-Willi syndrome (PWS) and Angelman syndrome can present autism symptoms, although the symptoms differ between the two conditions. According to the researchers, the findings have implications for treatment and intervention. Both Prader-Willi and Angelman syndrome are neurodevelopmental disorders that are…

A merger between OvaScience and Millendo Therapeutics will focus on advancing Millendo’s pipeline of treatments for orphan endocrine diseases (diseases involving hormones), including Prader-Willi syndrome (PWS), according to a press release. The merged company will operate under the name Millendo Therapeutics. “We are excited about the opportunities created by…

Patients with Prader-Willi syndrome (PWS) who develop significant obstructive sleep apnea and malocclusion can be successfully treated using surgical methods, according to a case report. The case study, “Orthognathic Correction in Prader-Willi Syndrome: Occlusion and Sleep Restored,” was published in The Cleft Palate-Craniofacial Journal. Prader-Willi children…

Adult patients with Prader‐Willi syndrome (PWS) show higher rates of psychiatric diagnoses that seem to be dependent on the type of genetic abnormality the patient has, a new study reports. The findings may help in the development of individualized therapeutic interventions. The study, “Prader‐Willi syndrome genetic subtypes and…

Soleno Therapeutics recently received fast track status from the U.S. Food and Drug Administration for diazoxide choline controlled-release (DCCR), its investigational treatment for Prader-Willi syndrome (PWS). DCCR is an extended-release, salt formulation of diazoxide, taken as a once-daily tablet. Diazoxide is a potassium channel activator, capable of crossing…

Inversago Pharma has raised $7 million to support the development of a new generation of cannabinoid-1 (CB1) receptor blockers for the treatment of Prader-Willi syndrome, type 1 diabetes, and other metabolic disorders. With the money, Inversago plans to advance its new compounds into clinical trials. CB1 inhibitors have…