Researchers have developed a more cost-effective supporting diagnostic test for hereditary angioedema (HAE) to detect genetic changes that are otherwise missed by current standard methods. The study, “Gene mapping strategy for Alu elements rearrangements: Detection of new large deletions in the SERPING1 gene causing hereditary angioedema in Brazilian…
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…
Asphyxiation caused by head and neck angioedema — swelling and fluid accumulation in the deeper layers of the skin — is rare, and most such deaths are due to bradykinin-mediated disease, a French study reported. In fact, bradykinin-mediated disease is 45 times more likely to be…
Takhzyro (lanadelumab), a kallikrein inhibitor by Shire, has been approved in Europe for the routine prevention of hereditary angioedema (HAE) attacks in patients ages 12 and older, making it the first antibody available in the EU for this indication. The European Commission’s decision comes shortly after the Committee for Medicinal Products…
Routine treatment with Shire‘s Takhzyro (lanadelumab) — given as a 300 mg dose every two weeks — significantly reduces the rate of acute attacks in hereditary angioedema (HAE) patients age 12 and older, a Phase 3 trial shows. The treatment also kept more patients attack-free during the 26-week study, reduced…
Long-term prevention with human formulations of the C1 esterase inhibitor — Cinryze or Haegarda — safely and effectively reduces the number of acute attacks in people with hereditary angioedema (HAE), but the therapies’ prices are too high, according to a report of the Institute for Clinical and Economic Review…
KalVista Pharmaceuticals’ investigational candidate KVD900, being developed for the treatment of hereditary angioedema (HAE), is safe and effectively inhibits kallikrein, the main driver of HAE attacks, results from a Phase 1 clinical trial show. Following these positive results, the company is now launching a larger Phase 2…
Delays between symptom onset and diagnosis of hereditary angioedema (HAE) types 1 and 2 have been decreasing, according to a real-world study that also revealed a younger age at diagnosis in this patient population over time. The study, “Improvement in diagnostic delays over time in patients…
The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), favors granting marketing authorization to lanadelumab for routine prevention of hereditary angioedema (HAE) attacks in patients ages 12 and older. Shire’s lanadelumab is a monoclonal antibody that specifically binds to…
With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit and affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate,…
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