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…
Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…
Scientists in Australia and Singapore are teaming up to investigate the usefulness of Cliniface, a 3D face analysis technology, to diagnose, treat, and manage patients with hereditary angioedema (HAE). The partnership, a collaboration between Takeda Global, SingHealth in Singapore, King Edward Memorial Hospital in Western Australia, Curtin University,…
Astria Therapeutics is gearing up to request permission for clinical testing in the U.S. of STAR-0215, its experimental therapy to prevent swelling attacks in hereditary angioedema (HAE). The company is expected to submit a formal request — in the form of an investigational new drug application — to the…
About one year of treatment with Takhzyro (lanadelumab) safely and effectively lowered the frequency of swelling attacks, while improving quality of life and treatment satisfaction in people with hereditary angioedema (HAE), according to interim data from the real-world EMPOWER study. In addition, nearly 1 in 5 patients was able to…
KalVista Pharmaceuticals has launched a Phase 3 trial, called KONFIDENT, to test the safety and effectiveness of KVD900, an experimental oral and on-demand treatment for swelling attacks in people with hereditary angioedema (HAE). “Beginning the KONFIDENT trial represents a major milestone for KalVista,” Andrew Crockett, the company’s…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
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