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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…
9 Meters Biopharma acquired global development rights to NM-136, a potential Prader-Willi syndrome (PWS) therapy, from its maker, Lobesity. The therapy candidate, formerly known as LOB-0136, aims to reduce obesity among PWS patients by targeting the glucose-dependent insulinotropic polypeptide (GIP), a molecule that helps sugar enter fat cells.
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
A protein that interacts with insulin growth factor 1 (IGF1) may be important in managing children with Prader–Willi syndrome (PWS) on growth hormone therapy, a study has found. The levels of that protein, called IGFBP7, were found to be higher than normal in PWS children who carry deletions in…
An additional clinical trial into diazoxide choline controlled release (DCCR) tablets as a potential treatment for the insatiable hunger associated with Prader-Willi syndrome (PWS) continues to be seen as necessary by U.S. Food and Drug Administration (FDA) to support a request for DCCR’s approval. But the agency also encouraged Soleno…
The U.S. Food and Drug Administration (FDA) has granted priority review to LV-101 (intranasal carbetocin), an investigational treatment for the distress behaviors and insatiable hunger that accompany Prader-Willi syndrome (PWS). The decision reduces the review period for the medication’s new drug application (NDA) to six months, down from the…
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