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…
KalVista Pharmaceuticals is planning to initiate a Phase 2 trial of its investigational oral therapy KVD824 as a preventive treatment for hereditary angioedema (HAE) attacks, the company announced. The decision follows promising data from two clinical trials, in which KVD824 was well tolerated and reached therapeutically relevant…
A single dose of NTLA-2002, Intellia Therapeutics’ investigational gene-editing therapy for hereditary angioedema (HAE), induces sustained reductions in the activity of plasma kallikrein — a protein involved in HAE swelling attacks — for at least 11 months, preclinical data shows. Based on these promising data in non-human…
In cases where targeted treatment for hereditary angioedema (HAE) is not available for rapid management of swelling attacks, patients may benefit from fresh frozen plasma (FFP) therapy, according to a recent case in Pakistan. Despite little information regarding best practices when using FFP, this success points to the…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions to…
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