Rare Disease Film Festival Highlights Patient and Researcher Unity
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 ... Read more
Updates
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BioCryst, Torii Partner to Commercialize BCX7353 in Japan to Prevent HAE Attacks
BioCryst Pharmaceuticals has licensed rights to Torii Pharmaceutical for the commercialization of BCX7353 — an oral, once-daily investigational therapy for the prevention of hereditary ... Read more -
Lupus Could Be Risk Factor for Angioedema, Study Suggests
Having systemic lupus erythematosus (SLE) can considerably increase the likelihood of developing angioedema, a new study suggests. Women, African-Americans, and younger patients may be particularly ... Read more -
Kalbitor Has Potential as Treatment for Angioedema Attacks in Lupus Teens Who Have Normal C1-IHN Levels, Case Reports Contend
Kalbitor (ecallantide) is a potential treatment for angioedema attacks that do not respond to medication, in adolescents who also have systemic lupus erythematosus (SLE), ... Read more
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Mutation Detection Method Cheaper, Quicker for HAE Diagnosis Than Sequencing, Study Says
A genetic technique called allelic discrimination — which detects a specific variant of the ... -
Short-term Use of Ruconest Can Prevent HAE Attacks Triggered by Medical or Dental Work, Study Reports
Short-term use of Ruconest (recombinant human C1 inhibitor) as a preventive treatment reduces the risk ... -
NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, ... -
NICE Recommends Takhzyro as Preventive Treatment for HAE Attacks
The National Institute for Health and Clinical Excellence (NICE) has recommended Takhzyro (lanadelumb) as ... -
Preventative Haegarda Therapy Quickly Reduces Number of HAE Attacks, Trial Analysis Shows
Prophylactic, or preventive treatment with Haegarda quickly reduced the number of attacks — sudden ... -
Pharvaris Raises $66M to Advance Oral Therapy for Hereditary Angioedema
Pharvaris reported raising $66 million in a second round of financing, allowing the company ...
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Stroke Patient Develops Orolingual Angioedema After Alteplase Administration, Case Study Reports
News
Researchers from University of Chicago Medicine reported the case of a patient who developed orolingual angioedema after the administration of alteplase (sold under the brand name Activase), and suggest how physicians should proceed in case of an emergency. The study, ... Read more