Favorable outcomes following early onset oral miglustat in early infantile Niemann Pick Type C

por Curelaru, Shiri, Zehavi, Yoav, Almagor, Tal, Spiegel, Ronen
Publicado 2021

A favorable outcome in an infantile-onset Pompe patient with cross reactive immunological material (CRIM) negative disease with high dose enzyme replacement therapy and adjusted im...

por Curelaru, Shiri, Desai, Ankit K., Fink, Daniel, Zehavi, Yoav, Kishnani, Priya S., Spiegel, Ronen
Publicado 2022