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Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights to…

Deletion mutations in the SERPING1 gene lead to lower function levels of the C1 inhibitor protein, which, in turn, might have a negative impact on the progression and severity of hereditary angioedema (HAE), a study reveals. The study, “Deletions in SERPING1 Lead to Lower C1 Inhibitor Function:…

Hereditary angioedema (HAE) can present itself as a sudden and severe abdominal pain, even in the absence of other typical symptoms, such as swelling in the skin and mucosal linings, a case study from Japan has found. According to the authors, physicians should not overlook these uncharacteristic symptoms and…

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, “A Case Report of Unilateral Orolingual Angioedema…

The U.S. Food and Drug Administration has rejected Pharming’s request to expand the label of Ruconest (conestat alfa) to include routine prevention of swelling attacks caused by hereditary angioedema (HAE). In a complete response letter indicating it had finished its review of the company’s supplemental biologics…

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

Medications that lower cholesterol levels, collectively known as statins, can in rare instances trigger angioedema episodes that are self-resolving if the patients stops using the statin, a case study reports. The study describes a side effect of Crestor (rosuvastatin), but its researchers caution that other, chemically-related statins could also…

Although the symptoms of hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE) present in childhood, most patients are diagnosed in adulthood, highlighting a need for increased awareness of C1-INH-HAE among pediatricians, according to a new study. The research, “Pediatricians diagnosed few patients with childhood-presented hereditary angioedema: Icatibant Outcome Survey…

Patients in Germany treat acute hereditary angioedema attacks with Firazyr (icatibant) earlier and have fewer severe attacks than do patients in 11 other countries where Firazyr is also approved, a retrospective study shows. The disparity is thought to arise from a easier access to Firazyr in Germany and a public healthcare…

The oral treatment candidate BCX7353 eases hereditary angioedema (HAE) attacks, reduces the occurrence of symptoms, and helps decrease the use of rescue medications, according to BioCryst Pharmaceuticals’ early Phase 2 trial results. The data also showed that the investigational therapy was safe and well-tolerated. BCX7353 is an oral…