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Gene therapies. Beremagene geperpavec (Vyjuvek): treatment of wounds. [ 2] Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [ 11] Lovotibeglogene autotemcel (Lyfgenia): treatment for sickle cell disease. [ 11]
KEGG. D11559. Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), [6][7] a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. [6] It works by providing a new copy of the SMN gene that produces the SMN protein.
The one-time therapy, branded as Lenmeldy in the U.S., is approved for children in certain stages of disease progression, the Food and Drug Administration (FDA) said. US approves first gene ...
The FDA approved Phase I clinical trials on thalassemia major patients in the US for 10 participants in July. [201] The study was expected to continue until 2015. [191] In July 2012, the European Medicines Agency recommended approval of a gene
The FDA's accelerated approval to Tecelra was based on data from a 44-patient study, in which 43.2% of the participants showed partial or complete response to the treatment for an average duration ...
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
CDD is caused by pathogenic variants in the gene CDKL5. This gene provides instructions for making a protein ( cyclin-dependent kinase -like 5) that is essential for normal brain development and function. [ 4] The CDKL5 protein is widely expressed in the brain, predominantly in nerve cells ( neurons ), with roles in cell proliferation, neuronal ...
The US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...