Sanofi’s Pompe Disease Therapeutic Now Accessible to NHS Patients

The drugmaker Sanofi announced that patients with Pompe disease, a rare disease that progressively damages the heart and muscles, will now have access to its long-term enzyme replacement therapy through the NHS. The drug was markedly more effective than the standard of care in preserving respiratory function than the current standard of care.

According to Emily Kimber, “Additionally, patients treated with Nexviadyme walked 32.2 metres further compared to baseline in the six-minute walk test, a key secondary endpoint, which was 30 metres further than the change with Myozyme.”

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(Source: PM Live, February 8^th, 2023)