Happy Friday! If you're observing Memorial Day, enjoy the upcoming long weekend. Going to use the time to brush up on a little rare disease news? We've got three articles…
According to Hemophilia News Today, data collected from a Phase 2b trial highlights that the subcutaneously administered DalcA is effective for the treatment of hemophilia B. Find the full press…
COVID-19 diagnoses are growing at a rapid rate, to the point where data is changing daily. As of today, there are 4.23 million diagnosed cases worldwide, with 1.39 million in…
As reported in WMS, Kathy Miska was beginning to have difficulty walking. This time, she knew what was happening. She'd seen the same thing happen to her sister, who went…
As reported in Biospace, Adverum Biotechnologies, a gene therapy company focused on clinical stage trials for rare and ocular diseases with unmet needs, has successfully dosed a patient in their…
According to Juvenile Arthritis News, treating juvenile idiopathic arthritis early helps reduce symptoms, nearly "inactivating" the condition. The Canadian Alliance of Pediatric Rheumatology Investigators (CAPRI) JIA Registry found that early…
The European Commission has just granted their approval to Pharming for an extended indication of RUCONEST (conestat alfa) for Hereditary Angioedema (HAE). This extension allows the therapy to be utilized for…
A New Study A new study, conducted by CLS Behring, has found that a new method of administration for Hizentra as a treatment for primary immunodeficiency (PI), could improve quality…
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