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Haegarda, a concentrated form of C1 inhibitor (C1-INH), is safe and effectively helps to prevent hereditary angioedema attacks in the long-term, allowing patients to become free of disease symptoms, That is the finding of a Phase 3 extension study, “Long-term outcomes with subcutaneous C1-inhibitor replacement therapy for prevention…

The three prophylactic medicines currently on the market for hereditary angioedema (HAE) — the C1 esterase inhibitors Cinryze and Haegarda, and Takzyhro (lanadelumab) — all reduce the number and severity of swelling attacks in patients with type 1/2 disease, without significant adverse events, a study found. But researchers report that these…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term…

There is a significant delay between symptom onset and an accurate diagnosis of hereditary angioedema (HAE) in Brazil, even in patients with a family history of the disease, according to a single-center study. Patients waited an average of 17.7 years from symptom onset to diagnosis, with one experiencing a…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Patients who experience an angioedema attack during treatment with angiotensin-converting enzyme inhibitors — a kind of medication for high blood pressure and heart failure — may safely switch to a different kind of blood pressure medication called angiotensin II receptor blockers, a study found. The study, “Angiotensin II receptor blockers…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

Using under-the-skin delivery of a C1-inhibitor (C1-INH) formulation in patients with hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE) decreased the number and severity of HAE attacks, was well-tolerated, and was associated with high patient satisfaction in a Phase 3 trial. The research, “Fixed-Dose Subcutaneous C1-Inhibitor Liquid…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders ranging…