A single dose of Ruconest (conestat alfa) was sufficient to rapidly and safely treat almost 96% of all acute attacks in a group of children with hereditary angioedema (HAE), Phase 2 trial data show. The treatment was approved for acute HAE attacks in adults and adolescents by the…
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the most determined. At that time, few doctors in the developing country of 2 million — now called North Macedonia — had…
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 problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…
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 of Virginia (iTHRIVE) to study a new biomarker for hereditary angioedema (HAE). iTHRIVE is awarding up to $50,000 to early-phase research projects…
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 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
People with hereditary angioedema appear to have a higher risk of developing non-alcoholic fatty liver disease, even before receiving angioedema medicines that affect liver function, a new study suggests. Researchers recommend that these patients undergo continuous monitoring to detect and manage fatty liver disease in its early stages. The…
People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…
Treatment with Takhzyro (lanadelumab) effectively reduces the number of attacks in patients with hereditary angioedema even in early phases of treatment, new analysis of data from the Phase 3 HELP study shows. Most recent trial results were shared in a poster, “Lanadelumab demonstrates rapid and sustained prevention of hereditary…
The unique mode each specific compound binds to and blocks the enzyme plasma kallikrein could lead to the development of improved therapies for patients with hereditary angioedema (HAE), according to a new study. The research, “Structures of full-length plasma kallikrein bound to highly specific inhibitors describe…
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