News

Higher blood levels of molecules involved in blood vessel formation and permeability — Tie-2, Ang-2, and VE-Cadherin — along with sE-selectin, which is involved in vascular inflammation, may help to distinguish different types of angioedema, a study suggests. Notably, Tie-2 may be a biomarker of hereditary angioedema (HAE),…

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats are also available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Takhzyro (lanadelumab) safely and markedly reduced the frequency of swelling attacks in people with hereditary angioedema (HAE) over 2.5 years, according to results of the HELP open-label extension (OLE) study. “This study supports the use of Takhzyro as a long-term preventative treatment option for those 12 years of…

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 into…

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…

The U.S. Food and Drug Administration (FDA) has approved a new convenience administration kit for people using Berinert, an at-home infusion treatment of acute hereditary angioedema (HAE) attacks in children and adults. This kit is designed to provide all the components needed for at-home treatment, making Berinert’s use easier…

RAPIDe-1, a Phase 2 clinical trial testing Pharvaris’ investigational oral therapy PHVS416 as an on-demand treatment for swelling attacks in people with hereditary angioedema (HAE), will expand its enrollment this year to include patients in the U.S. “We are encouraged by positive feedback from clinical sites and look forward…

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…

Effective management of hereditary angioedema (HAE) should include addressing psychological and mental health, given that depression, stress, and anxiety are “prevalent” among patients and can place a considerable burden on those with the chronic genetic disease, a study reported. Indeed, according to researchers, “all participants [in this study] experienced…

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…