Alfa-glucosidasa humana recombinante de leche de conejo en pacientes de Pompe

Lancet (London, England)
|September 6, 2000
PubMed
Resumen

El tratamiento temprano con alfa-glucosidasa humana recombinante (rhGAA) mejoró los síntomas de la enfermedad de Pompe en los bebés. Esta terapia enzimática normalizó la actividad enzimática, mejoró la función tisular y cardíaca y redujo el tamaño del corazón.

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