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…
Single, on-demand treatment with the investigational oral medicine KVD900 safely and significantly slowed the progression of swelling attacks and shortened the time to symptom relief and resolution in adults with hereditary angioedema (HAE), according to the latest data from a Phase 2 trial. “HAE patients continue to seek…
Treatment with Orladeyo (berotralstat) was generally safe and well-tolerated in people with hereditary angioedema (HAE), according to recently released trial data. The findings from an interim analysis of the open-label APeX-S trial also supported Orladeyo’s ability to reduce the frequency of HAE attacks, ease symptom burden, and improve…
Orchard Therapeutics and the Pharming Group are teaming up to research, develop, manufacture, and — possibly — commercialize a gene therapy for hereditary angioedema (HAE), called OTL-105. “This promising work toward treatment with the potential for durable long-term clinical benefit is encouraging and signifies an ongoing commitment to…
One in three patients with hereditary angioedema (HAE) may have an impaired sense of smell, but this does not appear to be tied to changes in the genes responsible for odor detection, a small study found. The scientists said more research is needed, in larger patient populations, to better…
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