News

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…

The Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), is recommending the approval of a prefilled syringe to administer Takhzyro (lanadelumab), a preventative treatment for hereditary angioedema (HAE) in patients 12 or older. The European Commission (EC), which typically accepts…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

After showing a sustained reduction in kallikrein levels in animal models, Intellia Therapeutics will be moving forward with the development of its gene-editing candidate NTLA-2002 for the treatment of hereditary angioedema (HAE). The company is now planning to ask regulatory authorities to advance the candidate into clinical studies…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…