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…
Pharming Group has signed an exclusive license agreement with NewBridge Pharmaceuticals to distribute and commercialize Ruconest (conestat alfa), a treatment for acute attacks of hereditary angioedema (HAE), in the Middle East and North Africa. Under the agreement terms, NewBridge in collaboration with Pharming, will provide access…
Multiple doses of PHA121 (PHA-022121), an investigational oral therapy for hereditary angioedema (HAE), were well-tolerated and rapidly reached therapeutic levels in healthy volunteers, according to data from a Phase 1 clinical trial. The therapy’s pharmacological profile “suggests that the therapeutic effect of PHA121 can be achieved as early…
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…
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