24], However, in 2012, a study of 94 patients with Pompe
disease represented a different pattern of muscle involvement.
The poster describes the results of an AAV gene therapy construct selection study conducted in a well-characterized mouse model of Pompe
disease that evaluated both AAV8 and AAV9 capsid serotypes, paired with novel promoter elements designed to target GAA expression in a range of tissues, including skeletal and cardiac muscle, the nervous system and the liver.
The poster presentation describes the results of an AAV gene therapy construct selection study conducted in a well-characterized mouse model of Pompe
Disease - Therapeutics under Investigation by Universities/Institutes 13
I feel that Pompe
opened up the field," Kishnani said.
disease "is characterized by delayed motor skills (such as rolling over and sitting) and progressive muscle weakness," according to NIH's description of the disease.
The Phase III trial is a randomized, multi-center, multi-national, double-blinded study to compare the efficacy and safety of repeated bi-weekly infusions of neoGAA and alglucosidase alfa in treatment-naive patients with late-onset Pompe
Por ejemplo, un participante reflexionaba: El pompe
era la imagen de este objeto pintado de rojo, ustedes nombraron como pompe
The report provides a snapshot of the global therapeutic landscape of Pompe
In 2006, the FDA approved an enzyme replacement therapy called Myozyme (Alglucosidase alfa, rhGAA), for people with Pompe
The new product candidate, designated AT003, expands Audentes' portfolio of investigational AAV gene therapy treatments for rare diseases with significant unmet medical needs, which also includes AT001 for X-Linked Myotubular Myopathy (XLMTM) and AT002 for Pompe
com)-- Access Health[R], a magazine-style health and wellness series, will dedicate an entire 30-minute episode to raising awareness about Pompe
Disease on Wednesday, April 15 at 7:30 a.
Schwarz has experience counseling clients, providing opinions and working on cases involving a range of pharmaceutical and biotechnology products, including drugs or treatments for renal disease, HCV, Pompe
disease and schizophrenia; modified dosage forms; drug delivery devices; and polymers.
He has Pompe
disease, which gives him minimal use of his muscles.
disease is so rare that few doctors in the world knew about it.