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Pitolisant, a treatment for narcolepsy, should be considered for people with Prader-Willi syndrome (PWS) whose response to psychotropic medications is less than optimal, a case report of a 15-year-old patient suggests. “Pitolisant has the potential to improve many symptoms in patients with Prader-Willi syndrome and it appears to be…
The hunger hormone ghrelin, when measured in infancy, may be a biomarker for predicting the early onset of scoliosis — a sideways curvature of the spine that looks like the letter C or S — in children with Prader-Willi syndrome (PWS), a study suggested. Beginning in early infancy,…
Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…
Aardvark Therapeutics has raised $29 million in financing to help support three planned Phase 2 clinical trials of its lead compound, ARD-101, an investigational therapy for Prader-Willi syndrome (PWS) and other disorders. Financing was led by Sorrento Therapeutics, with participation from other investors and support from the…
Growth hormone treatment is safe and helps to increase lean body mass and reducing body fat in adults with Prader-Willi syndrome (PWS), according to an analysis of published studies. Findings also suggest that such improvements in body composition might lower the risk of cardiovascular problems in adults with the…
More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…
Radius Health plans to initiate a Phase 2/3 study to evaluate the safety and efficacy of its investigational therapy RAD011, a lab-made oral cannabidiol therapy to treat excessive appetite, or hyperphagia, in people with Prader-Willi syndrome (PWS). The study, called SCOUT-015, is expected to begin by the…
The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
Twice daily supplementation with a probiotic may help reduce body mass index (BMI), a measure of body fat, in patients with Prader-Willi syndrome (PWS), a clinical trial in China has found. The probiotic — Limosilactobacillus reuteri LR-99 — also improved social communication and interaction, fine motor skills, and overall development…
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