
Great research news from @UCSFcmfpm and @MackenzieTippi — case study on in utero enzyme replacement treatment of a fetus with infantile-onset Pompe—a lysosomal storage disease.
NEJM@NEJM
Infantile-onset Pompe’s disease is caused by a genetic deficiency of an enzyme critical to heart development. This case study involved the intrauterine treatment of an affected fetus with enzyme replacement at approximately 6 months of gestation. nej.md/3NM3Avl
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