We use cookies to help provide you with the best possible online experience. Please read our Privacy Policy for information about which cookies we use and what information we collect on our site. By continuing to use this site, you agree that we may store and access cookies on your device.
Patients with late-stage Pompe disease who are failing to respond to enzyme replacement therapy will now have early access to an alternative treatment option, after Amicus Therapeutics' cipaglucosidase alfa with miglustat was approved for the UK's ...