KVD900, a potential new approach for treating hereditary angioedema, prevents the breakdown of kininogen and subsequent buildup of bradykinin — the underlying cause of the disease, KalVista Pharmaceuticals announced. The company confirmed this mechanism of action using a new test that measures the amounts of high molecular weight kininogen in the plasma. The…
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As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…
Entresto (sacubitril/valsartan) — an approved medication for heart failure — does not significantly increase the risk of angioedema compared with standard therapy Vasotec (enalapril), data from a Phase 3 trial show. The medication was also better at preventing deaths and heart failure-related hospitalizations than Vasotec, suggesting it could…
Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…
The European Medicines Agency issued a positive opinion on BioCryst Pharmaceuticals’ application for orphan drug status of BCX7353 to treat hereditary angioedema (HAE). The European Commission is expected to adopt the recommendation from the EMA’s Committee for Orphan Medicinal Products in the next 30 days. With orphan drug designation, BCX7353 will receive…
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Human Plasma-derived C1-INH Can Help Manage Hereditary Angioedema During Pregnancy, Study Reports
Treatment with human plasma-derived C1-INH is an effective and safe option for managing symptoms of severe hereditary angioedema during pregnancy and delivery, a Swedish case report suggests. The study, “Management of Women with Hereditary Angioedema During Pregnancy and Delivery,” was published in the Journal of Anesthesia, Surgery and…
The most common type of angioedema mediated by bradykinin is caused by the use of common blood pressure medications called angiotensin-converting enzyme inhibitors, a review study shows. This is followed by hereditary angioedema and recurrent angioedema, occurring due to an acquired C1-inhibitor deficiency, researchers say. The study, “…
Lisinopril, a medication widely used to treat high blood pressure and other cardiovascular disorders, sometimes may trigger small bowel angioedema, suggests a case report. While rare, physicians should recognize angioedema as a possible side effect of lisinopril, and provide timely diagnosis and adequate treatment in the presence of acute abdominal pain.
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD), held…
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