Self-made hematopoietic stem cell gene therapy OTL-203 for the treatment of type I muscosal polysaccharides: fda-named orphan medicine and rare pediatric disease.

It was announced that its in vitro hematopoietic stem cell () gene therapy-type mucosacized reservoir disorder () was awarded the title of Orphan Drug and the title of Rare Pediatric Disease by the U.SFood and Drug AdministrationPhoto Source: A temporary data trial was recently announced to assess the safety and effectiveness of the experimentAll eight patients treated reached the primary endpoint of the trial, and in the first two patients who followed at least one year, motor skills improved cognitive performance and normal growth compared to baseline levelsAbout heterolyse polysaccharides () is a rare hereditary neurometabolic disease caused by the lack of alpha-aduaform acid asse () lysozyme () this enzyme needs to break down a sugar molecule called glycosamine polysaccharides (also known as glycosain)Accumulation in multiple organs can cause a variety of symptoms including neurocognitive impairment, bone deformities, vision and hearing loss, and cardiovascular and lung complicationsThe overall estimated frequency of occurrence is one for each infantThere are three seed types about % of children with the most severe subtype called syndrome without treatment rarely survivingTreatment options, including hematopoietic stem cell transplantation and chronic enzyme replacement therapy, have obvious limitationsAlthough early intervention with enzyme replacement therapy has been shown to delay or prevent certain clinical features of the disease, the efficacy of neurological symptoms is limitedAn in vitro hematopoietic stem cell gene therapy is currently being studied and treatedThe exclusive worldwide license for intellectual property was developed by the Italian Institute of Gene Therapy