Nature: Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV ...
Pompe disease results from lysosomal acid α-glucosidase (GAA) deficiency and its associated glycogen accumulation and muscle damage. Alglucosidase alfa (recombinant human GAA (rhGAA)) received ...
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV ...
Pompe disease is a rare genetic disorder that’s caused by an inability to break down glycogen, a form of sugar that the body stores in the muscles for energy. As glycogen accumulates in toxic ...
FiercePharma: Sanofi notches another Pompe win with FDA blessing for enzyme replacement therapy Nexviazyme
In the realm of rare diseases, Sanofi has added another arrow to its Pompe quiver. The FDA cleared Sanofi’s Nexviazyme, also known as avalglucosidase alfa-ngpt, to treat patients ages one and older ...
Sanofi notches another Pompe win with FDA blessing for enzyme replacement therapy Nexviazyme