Ruconest Can Safely and Effectively Treat Acute HAE Attacks in Children, Phase 2 Trial Shows
A single dose of Ruconest (conestat alfa) was sufficient to rapidly and safely treat almost 96% of all acute attacks in a group of ... Read more
Updates
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Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have ... Read more -
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing ... Read more -
iTHRIVE to Fund Early Research Projects, Including Hereditary Angioedema
Researchers from the University of Virginia School of Medicine and Inova Health System have received seed grant funding from the Integrated Translational Health Research Institute ... Read more -
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the ... Read more
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Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ... -
Hereditary Angioedema Patients Have High Incidence of Fatty Liver Disease, Study Suggests
People with hereditary angioedema appear to have a higher risk of developing non-alcoholic fatty liver ... -
Takhzyro Induces Rapid Resolution of Hereditary Angioedema Attacks, New Data Analysis Shows
Treatment with Takhzyro (lanadelumab) effectively reduces the number of attacks in patients with hereditary angioedema ... -
Method of Blocking Kallikrein Could Help Develop Better HAE Therapies, Study Suggests
The unique mode each specific compound binds to and blocks the enzyme plasma kallikrein ... -
KVD900, KalVista’s Investigational Therapy for HAE, Fares Well in Phase 1 Trial
KalVista Pharmaceuticals‘ investigational therapy KVD900 for the treatment of hereditary angioedema (HAE) rapidly reduces the ...
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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