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…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery and…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
KVD900, an oral experimental on-demand treatment for swelling episodes caused by hereditary angioedema (HAE), safely and significantly reduced the severity of attacks and shorted the time to improvement, which was maintained for 24 hours, according to Phase 2 trial data. “Our goal is to provide the best outcome…
The Israeli Ministry of Health has agreed to review, under an accelerated process, a marketing application for Orladeyo (berotralstat), BioCryst Pharmaceuticals’ oral preventive treatment for hereditary angioedema (HAE) attacks, in patients 12 and older. Neopharm Israel, one of the leading pharmaceutical companies in Israel, will distribute Orladeyo,…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
Spain has added Ruconest (conestat alfa), Pharming’s treatment for acute attacks of hereditary angioedema (HAE), to its list of reimbursable medications. “We are delighted with this positive reimbursement decision by the Spanish Ministry of Health, as it means patients in Spain in need of new treatment options for…
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