The US Food and Drug Administration has approved a drug called TakhzyroTM (lanadelumab-flyo) for the treatment of hereditary angioedema. This approval was based on supporting evidence from several studies, including from…
According to a story from globnewswire.com, the gene therapy company Adverum Biotechnologies recently announced that the US Food and Drug Administration (FDA) has granted them Orphan Drug designation for their…
According to a story from Pharma Times, four new medications were recently approved for coverage on the Scottish NHS by the Scottish Medicines Consortium (SMC). Some of these drugs are…
According to a story from Benzinga, the pharmaceutical company BioCryst Pharmaceuticals, Inc., recently announced that its investigational product BCX7353 has been granted Fast Track Designation from the FDA for the…
As I’ve gotten older, I realize I’ve become more introspective, analyzing my relationships, emotions, spiritual well being, and mental health. While talking to others with rare diseases I’ve learned it…
The information in this article is based on the HAE UK and the US Hereditary Angioedema Association websites. Hereditary angioedema (HAE) is a condition that affects about one in every…
Researchers have carried out a study into the genetic mechanisms involved in hereditary angioedema acute attacks. They found that two genes, called uPAR and ADM, had a particularly strong influence.…
It may not be surprising news to find that the United States now pays more for prescription drugs than any other high-income country. This was not always the case however. Read…
According to a story from Rare Disease Report, a recent study indicates that the self-administration of Berinert, a drug used to treat hereditary angioedema, is effective and safe for patients…
Have you read part one of Loukisha's story? Check it out here! When I imagined life with my husband and children my plans didn’t include surgeries, walkers, canes, a wheelchair,…
From birth until my mid-thirties I was a very active person. I loved dancing West Coast Swing, doing yoga, and cooking large meals for my ‘Ohana. The word "'Ohana" describes…
According to a recent story from pm360online.com, the pharmaceutical company Shire announced that the marketing application for its drug product lanadelumab has gained approval from the European Medicines Agency (EMA).…
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