Takeda’s Takhzyro (lanadelumab) continues to safely prevent and reduce the frequency of swelling attacks in people with hereditary angioedema (HAE), according to 2.5 years of data from the HELP open-label extension (OLE) trial. The study’s final results were presented in two company-sponsored posters at the 2020…
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
Takeda Canada announced reaching an agreement for public funding in Canada of Takhzyro (lanadelumab), its treatment for eligible hereditary angioedema (HAE) patients. The agreement was reached between the company and the pan-Canadian Pharmaceutical Alliance, the agency responsible for setting the prices of brand and generic medications available throughout…
Berotralstat — an oral therapy by BioCryst Pharmaceuticals for preventing swelling attacks in people with hereditary angioedema (HAE) — will be available to patients, 12 and older, in the U.K. through an Early Access to Medicines Scheme (EAMS) before its potential regulatory approval. An EAMS gives patient access to…
Preventive treatment with oral berotralstat (BCX7353) significantly lowers the frequency of swelling attacks in people with hereditary angioedema (HAE), newly published results from the APeX-2 clinical trial demonstrate. The study, “Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: a randomized, double-blind, placebo-controlled phase 3…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
The first documented case of a hereditary angioedema (HAE) attack in an unborn child — whose symptoms resolved after treating the mother with the recombinant human C1-inhibitor (rhC1-INH) therapy, Ruconest — has been reported. The case report, “Hereditary Angioedema Attack in Utero and Treatment of the Mother…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,” the…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
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