Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening ... Read more
Updates
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FDA Agrees to Review Berotralstat as Preventive Treatment for HAE Attacks
The U.S. Food and Drug Administration (FDA) agreed to review BioCryst Pharmaceuticals‘ application requesting approval of oral berotralstat as a once-daily treatment to prevent ... Read more -
Ruconest Safe, Effective for On-demand and Preventive Treatment of HAE Attacks, Study Says
Ruconest (rhC1INH) is safe and effective as an on-demand therapy to treat acute swelling attacks, and as a short-term prophylaxis to prevent attacks of hereditary ... Read more -
Study Identifies Novel Molecular Players as Potential Therapeutic Target in Bradykinin-mediated Swelling Attacks
Bradykinin, a key molecular player involved in swelling attacks in hereditary angioedema patients, does so by signaling through the cyclooxygenase (COX) proteins, which may ... Read more -
Intellia Presents First Data Supporting CRISPR/Cas9 Gene Therapy Candidate for HAE, Anticipated to Lessen Angioedema
Intellia Therapeutics announced the first preclinical results supporting a gene therapy candidate for hereditary angioedema (HAE). In animal models, the product markedly and sustainably ... Read more
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Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day ... -
Preventative Therapy for HAE Attacks, Haegarda, Now Available in Quebec
Haegarda, a C1-INH replacement medication by CSL Behring, is now available in the Canadian ... -
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 ... -
Life Expectancy Similar for HAE Patients and General Population in Italy, Study Shows
In Italy, people with hereditary angioedema (HAE) types 1 or 2 are not at ... -
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan ... -
BioCryst Seeks Berotralstat Approval in Japan for Prevention of HAE Attacks
BioCryst Pharmaceuticals has submitted an application to the Japanese regulatory authority asking that oral ...
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Stroke Patient Develops Orolingual Angioedema After Alteplase Administration, Case Study Reports
News
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, ... Read more