Please provide your email address to receive an email when new articles are posted on . Meningococcal vaccination rates are low among eligible patients with complement component deficiencies, ...
A single treatment with a CRISPR-Cas9 based gene editing therapy is enough to replace the daily medication of patients with hereditary angioedema (HAE), a condition characterized by severe, painful ...
The fact that the complement system is activated during immune-complex glomerular disease has been known for nearly 50 years. Detection of complement deposition in the glomerulus using immunochemistry ...
Philadelphia and Gordonville, PA, August 12, 2025 – Researchers from Children’s Hospital of Philadelphia (CHOP) and the Clinic for Special Children found that complement factor I (CFI) deficiency, an ...
Researchers from Children's Hospital of Philadelphia (CHOP) and the Clinic for Special Children found that complement factor I (CFI) deficiency, an ultra-rare genetic disorder that can cause ...
Studies of monogenic gastrointestinal diseases have revealed molecular pathways critical to gut homeostasis and enabled the development of targeted therapies. We studied 11 patients with abdominal ...
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced interim results from a Phase 1/2a clinical study of ARO-CFB, the company’s investigational RNA ...
A deficiency disease is a medical condition caused by insufficient intake of essential nutrients, such as vitamins and minerals, that the body needs to function properly. These nutrients are normally ...
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