A collaborative research team successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy from 4s3 Bioscience.
Enzyme Replacement Therapy for Fabry Disease may be Improved by Inventive Approach
A creative structure-based remodeling strategy to design a therapeutic protein for Fabry disease has been used in a new study
A creative structure-based remodeling strategy to design a therapeutic protein for Fabry disease has been used in a new study
Michael W. Lawlor, M.D., Ph.D., assistant professor of pathology at MCW, researcher at the Children's Hospital of Wisconsin Research Institute, and director of the pediatric pathology neuromuscular laboratory in MCW's division of pediatric pathology, coordinated a study at Boston Children's Hospital and MCW that used targeted enzyme replacement therapy to deliver myotubularin to muscles of mice with XLMTM. After two weeks of treatment, the mice showed marked improvement in muscle function and pathology.
"These promising findings suggest that even low levels of myotubularin protein replacement can not only improve weakness in patients, but also at least partially reverse the structural abnormalities seen in XLMTM," said Dr. Lawlor. "The next step is to determine appropriate dosage, and toxicity, before we venture into human trials," he continued.
Congenital Form of Rickets Could Be Treated by Enzyme Replacement Therapy
In a study on mice, researchers at the Burnham Institute for Medical Research have successfully discovered that enzyme replacement therapy may act as a potential treatment for preventing
In a study on mice, researchers at the Burnham Institute for Medical Research have successfully discovered that enzyme replacement therapy may act as a potential treatment for preventing
Study Identifies Genes Linked to Stuttering
Stuttering, a speech problem that stigmatizes millions worldwide and has stumped scientists for centuries, could be caused by defects in three genes, a landmark study showed this week.
Stuttering, a speech problem that stigmatizes millions worldwide and has stumped scientists for centuries, could be caused by defects in three genes, a landmark study showed this week.
Enzyme Therapy may Slow Deterioration of Kidney Function
Enzyme replacement therapy (ERT) with agalsidase alfa may slow deterioration of kidney function in men with Fabry disease, provided the treatment is started early, suggests a new study.
Enzyme replacement therapy (ERT) with agalsidase alfa may slow deterioration of kidney function in men with Fabry disease, provided the treatment is started early, suggests a new study.
