Preventive treatment with Haegarda, an under-the-skin C1-INH replacement medication by CSL Behring, significantly lowers the ...

Bridge Medicines and The Rockefeller University are collaborating to treat hereditary angioedema (HAE) by developing ...

The Phase 2 clinical trial testing KalVista Pharmaceuticals‘ oral investigational therapy KVD900 as an on-demand treatment for hereditary angioedema (HAE) ...

Short-term preventive treatment with Berinert, a plasma-derived form of the C1 inhibitor protein, protects people with ...

Antisense oligonucleotides (ASOs) being developed by Ionis Pharmaceuticals may reduce the frequency of attacks in ...

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is ...

A case report suggests that the body has multiple ways to produce bradykinin, a molecule ...

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed ...

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to ...

BioCryst Pharmaceuticals is prepared to launch Orladeyo (berotralstat), an oral treatment for hereditary angioedema (HAE), ...

More insight and study is needed into the symptoms, proper management, and potential complications of ...

KalVista Pharmaceuticals anticipates beginning a Phase 2 clinical trial in the second half of this ...

HAE International, a global nonprofit that supports local hereditary angioedema (HAE) patient groups and organizations, ...

Cipla has received final approval from the U.S. Food and Drug Administration (FDA) for a generic ...

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease ...

Preventive treatment with Takhzyro (lanadelumab) significantly reduces the number of swelling attacks in people with hereditary angioedema ...

Regular Haegarda treatment in older people with hereditary angioedema (HAE) — those ages 65 and ...

Preventive treatment with Takhzyro (lanadelumab) is effective at delaying and lowering the monthly rate of ...

Ruconest, a recombinant human form of the C1 inhibitor protein, is more cost-effective than other ...

Preliminary results from a Phase 2 clinical trial testing the investigational therapy garadacimab for the ...

New clinical trial data show that BioCryst Pharmaceuticals‘ investigational therapy for hereditary angioedema (HAE), berotralstat, ...

The U.S. Patent and Trademark Office (USPTO) issued a new allowance for a patent submitted ...

The Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency ...

Costs are unsustainable for preventive treatment of hereditary angioedema (HAE) attacks with approved therapies Takhzyro or Haegarda, ...

The European Commission has approved Pharming‘s Ruconest (conestat alfa) for the treatment of acute swelling episodes ...

A small molecule inhibitor being developed for hereditary angioedema (HAE) treatment, called PHA121, was as effective ...

The European Medicines Agency has validated BioCryst Pharmaceuticals‘ application requesting the approval of berotralstat as a ...

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended Ruconest (conestat ...

The U.S. Food and Drug Administration (FDA) has approved a new production facility for Ruconest (conestat ...

The number of treatments for children with rare diseases has grown over the past decade, according ...

In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign ...

Mutations in the KCNMA1 gene may predispose certain people to angioedema caused by blood pressure medications, ...

A new diagnostic test for hereditary angioedema (HAE) can measure the activity of C1-inhibitor (C1-INH) ...

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles ...

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave ...

The U.S. Food and Drug Administration (FDA) agreed to review BioCryst Pharmaceuticals‘ application requesting approval ...