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…
PHA121 (PHA-022121), Pharvaris’ experimental oral therapy for hereditary angioedema (HAE), results in faster, more potent, and more durable suppression of bradykinin-induced changes in blood pressure than injectable Firazyr (icatibant), according to studies in monkeys and healthy volunteers. These findings, along with evidence of PHA121’s favorable pharmacological…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
Orladeyo (berotralstat), an oral therapy to prevent hereditary angioedema (HAE) attacks, is now available to patients, 12 and older, in Germany. “We have an experienced team in place in Germany that is excited and honored to bring the first oral, once daily therapy to HAE patients in Europe,”…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in grants, totaling up…
A hereditary angioedema (HAE) patient with a rare gene mutation underwent several rounds of treatment adjustments and experienced multiple side effects while transitioning to off-label Takhzyro (lanadelumab‐flyo) over a period of 12 weeks, a study reports. …
The U.S. Food and Drug Administration (FDA) has approved a new generic version of Firazyr (icatibant injection), a medication to treat acute swelling attacks in patients with hereditary angioedema (HAE), Glenmark Pharmaceuticals, announced in a press release. According to the India-based company, the newly approved generic will be…
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