For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

The FDA approved the enzyme replacement therapy Adzynma for the prophylactic or on-demand treatment of adults and pediatric patients with congenital thrombotic thrombocytopenic purpura, a rare blood ...

Adzynma is a purified recombinant form of the ADAMTS13 protein designed to replace the missing or deficient ADAMTS13 enzyme. The Food and Drug Administration (FDA) has approved Adzynma for ...

Adzynma is the first product to be approved for the treatment of the rare blood disorder that has 90% mortality when left untreated. Congenital thrombotic thrombocytopenic purpura (cTTP) can now be ...

CHARLOTTE, N.C. — Prophylactic endotracheal intubation prior to endoscopy was not beneficial for patients with upper gastrointestinal bleeding and was instead tied to adverse risks, researchers ...

Prophylactic cardiac treatment in DMD patients is associated with a 54% lower hazard of death, highlighting its potential survival benefit. Only 27.7% of DMD patients received prophylactic cardiac ...

A mucus-based prophylactic gel developed at KTH Royal Institute of Technology has proven 70 percent effective in lab tests against HIV, and 80 percent effective against herpes. The viral prophylactic ...

Current guidelines recommend 5 to 7 days of broad-spectrum antibiotic prophylaxis for upper gastrointestinal bleeding in patients with cirrhosis. In a systematic review and meta-analysis, shorter ...

HEMGENIX®, the first and only FDA approved gene therapy for hemophilia B, has been proven to elevate and sustain factor IX levels for years, significantly reduce the rate of annual bleeds versus ...

KalVista Pharmaceuticals' share price has been cut in half after the company scrapped one of its hereditary angioedema programs in the face of multiple reports of raised enzyme levels. Elevated ALT ...