News

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

Preventive treatment with Takhzyro (lanadelumab) significantly reduces the number of swelling attacks in people with hereditary angioedema (HAE), even in the early phases of treatment, a post hoc analysis from the HELP trial shows. The benefits are evident within two weeks of treatment and progressively increase over time, the researchers…

Regular Haegarda treatment in older people with hereditary angioedema (HAE) — those ages 65 and older — was well-tolerated and effective in stopping HAE-related attacks, a post-hoc analysis of the COMPACT Phase 3 clinical trial revealed. The therapy was found to be well-tolerated…

Preventive treatment with Takhzyro (lanadelumab) is effective at delaying and lowering the monthly rate of attacks in a broad range of patients with hereditary angioedema (HAE), a study has found. The therapy did so by inducing sustained increases in blood concentrations of the treatment. The response was not…

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the…

Ruconest, a recombinant human form of the C1 inhibitor protein, is more cost-effective than other on-demand therapies at treating attacks in people with hereditary angioedema, a new study suggests. The study, “Modeling Cost-Effectiveness of On-Demand Treatment for Hereditary Angioedema Attacks,” was published in the …

Preliminary results from a Phase 2 clinical trial testing the investigational therapy garadacimab for the treatment of hereditary angioedema (HAE) show a more than 88% reduction in the number of attacks, the therapy’s developer CSL Behring announced.  The…

New clinical trial data show that BioCryst Pharmaceuticals‘ investigational therapy for hereditary angioedema (HAE), berotralstat, reduces the frequency of HAE attacks and improves patients’ quality of life through 48 weeks of treatment. The data were presented at the European Academy of Allergy and Clinical Immunology Digital Congress. HAE…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

The U.S. Patent and Trademark Office (USPTO) issued a new allowance for a patent submitted by BioCryst Pharmaceuticals for berotralstat (BCX7353), an investigational therapy to treat hereditary angioedema (HAE). The USPTO issued a notice of allowance to BioCryst, indicating future approval. Once approved, the new “composition…