News

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

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…

Growth Hormone Therapy Safe and Helpful for PWS Adults, Study Finds

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…

Advocates Lobby US Congress During Virtual Rare Disease Week

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…

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

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…

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

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…

This Probiotic May Help Prevent Obesity in Children with PWS

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…