Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder that can lead to lung and liver disease. Individuals with AATD have a deficiency of a protein called alpha-1 antitrypsin (AAT), which normally protects the lungs and liver from damage caused by enzymes. The treatment approach for AATD often involves managing symptoms and providing supportive care.
Latest Alpha-1 Antitrypsin Deficiency Treatment Companies Update
Nov 2023: One of the top manufacturers of plasma-derived medications and cutting-edge diagnostic tools worldwide, Grifols, today announced the release of its new Grifols sCD38 solution, the first soluble recombinant protein in the industry created to block anti-CD38 antibodies in patients with multiple myeloma undergoing daratumumab therapy. This guarantees prompt and precise blood transfusion tests essential for appropriate treatment. A monoclonal antibody directed against CD38 called daratumumab treats multiple myeloma. Nevertheless, the medication attaches to red blood cells CD38 protein during therapy, which may change the outcomes of vital blood pre-transfusion testing. For certain individuals, this may cause a delay in vital transfusions. Grifols' DG Gel technology for pre-transfusion compatibility testing makes screening, identification, and crossmatching more efficient thanks to Grifols sCD38's unique methodology.
Nov 2023: Takeda's Adzynma (recombinant-krhn ADAMTS13) has been permitted by the US Food and Drug Administration to treat congenital thrombotic thrombocytopenic purpura (cTTP), a rare blood clotting condition. Adzynma is recommended for use in adult and pediatric cTTP patients as an on-demand and preventative treatment. Approved for this use, it is the only recombinant A disintegrin and metalloproteinase with thrombospondin motifs 13 protein. This protein therapy functions by substituting the ADAMTS13 enzyme that is defective. The most recent advancement is predicated on results from a Phase III clinical trial and continuation study that was open-label, controlled, randomized, crossover. In patients with cTTP, the Phase III trial assessed the safety, effectiveness, tolerability, and pharmacokinetics of intravenous Adzynma dosages compared to plasma-based treatments.
Alpha 1 Antitrypsin Deficiency Treatment Market Highlights:
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