The one-year Phase 3 SPRING study evaluating the safety, efficacy, and pharmacological properties of Takhzyro (lanadelumab) in children with hereditary angioedema (HAE), ages 2 to 11, succeeded in meeting its goals, Takeda, which markets the treatment, announced. Based on these findings, the company plans to file applications…
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…
Six warning signs were present in a group of patients with hereditary angioedema (HAE) due to a deficiency in C1-inhibitor (C1-INH), as reported in a recent study from Brazil. Dubbed H4AE to assist in recalling, the warning signs are hereditary (H), recurrent angioedema (A), abdominal pain (A), absence of…
Takhzyro (lanadelumab) led to significant benefits for patients with hereditary angiodema (HAE) in France who received the therapy as part of an early-access program prior to its approval in Europe, a study has found. The treatment “reduced attack rates, improved quality of life, and was generally well tolerated,”…
Patients with hereditary angioedema (HAE) are more likely to develop comorbidities, or co-existing conditions, such as cardiovascular and autoimmune diseases than the general population, a Swedish database study reports. The findings highlight the need for awareness and prevention of these conditions in patients with HAE, according to researchers. The…
Takhzyro (lanadelumab) has been approved in Japan to prevent acute swelling attacks in people, 12 and older, with hereditary angioedema (HAE). An estimated 2,000 to 3,000 people with HAE live in Japan, but fewer than 500 have been diagnosed due to a lack of awareness, according to…
Strategies to manage discontinuing attenuated androgens — male hormones frequently used as a preventive treatment for hereditary angioedema (HAE) — can be very diverse, and the most common withdrawal challenge is an increase in HAE attacks, a recent case series reported. Its authors, some of which are leading European…
Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…
A 14-year-old boy developed angioedema of the tongue thought to be triggered by Haldol (haloperidol), an antipsychotic medication used to treat schizophrenia and other mental or mood disorders, a case report found. “Clinicians should be aware of potential dangerous adverse effects of commonly used medications. Patients with angioedema may…
Donidalorsen significantly reduced swelling attacks and improved quality of life in people with hereditary angioedema (HAE) treated in a Phase 2 trial. Full trial results were detailed in the study, “Inhibition of Prekallikrein for Hereditary Angioedema,” published in The New England Journal of Medicine. The study…
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