What is Onasemnogene abeparvovec-xioi Intravenous
Onasemnogene abeparvovec-xioi is a gene therapy drug used to treat spinal muscular atrophy (SMA), a rare genetic disorder that leads to muscle weakness and loss. It is a genetically modified virus that delivers a healthy copy of the SMN gene to cells in the body, helping to improve muscle function and extend life expectancy. Onasemnogene abeparvovec-xioi is given intravenously, typically once every four weeks.
Benefits of Onasemnogene abeparvovec-xioi Intravenous
and Its Uses.
Onasemnogene abeparvovec-xioi (trade name Zolgensma) is a gene therapy drug that was approved by the United States Food and Drug Administration (FDA) in May 2019 for the treatment of spinal muscular atrophy (SMA) in patients aged 2 years and older. SMA is a rare, genetic neuromuscular disorder that results in the progressive loss of muscle function and is the leading genetic cause of death in infants and children.
Onasemnogene abeparvovec-xioi is a genetically modified virus that delivers a functional copy of the SMN1 gene to cells that are affected by SMA. The SMN1 gene is responsible for the production of a protein called survival of motor neuron 1 (SMN), which is essential for the survival of motor neurons. The delivery of the SMN1 gene by onasemnogene abeparvovec-xioi allows the
Side Effects and Dosage of Onasemnogene abeparvovec-xioi Intravenous
Onasemnogene abeparvovec-xioi is a gene therapy drug that is used to treat patients with hemophilia A. It is a recombinant adeno-associated virus (rAAV) that contains the gene for Factor VIII (FVIII), which is deficient in patients with hemophilia A. The rAAV is administered intravenously, and the FVIII gene is expressed in cells throughout the body, including the liver, which produces Factor VIII protein.
Onasemnogene abeparvovec-xioi is generally well tolerated. The most common side effects are headache, fever, and chills. These side effects are usually mild and resolve without treatment.
Onasemnogene abeparvovec-xioi is a prescription drug and should be used only under the supervision of a healthcare provider. The recommended dosage depends on the weight and age of the patient. The dose is typically 2