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…
Pharma and patient organizations came together this year in celebration of the 10th annual HAE Awareness Day — set aside to call global attention to hereditary angioedema (HAE). Set annually on May 16, this event aims to increase awareness among the public, healthcare professionals, healthcare decision-makers, and industry…
Following a minor surgical procedure, a 70-year-old Indian man developed angioedema of the tongue that was likely triggered by an ACE inhibitor — a class of medicines often used to manage high blood pressure — a case report found. The patient had a…
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