Cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease: PROPEL study subgroup analyses
Presenter: Mark Roberts, MD, Consultant Neurologist, Salford Royal NHS Foundation Trust, Greater Manchester Neurosciences Centre, United Kingdom (ePoster #196)
Living with late-onset Pompe disease in the UK: interim results characterising the patient journey and burden on physical, emotional and social quality of life
Presenter: Alan Muir, Chair of the Board of Trustees, Pompe Support Network, United Kingdom (ePoster #201)
Cipaglucosidase alfa/miglustat is an investigational therapy that is not yet approved by any regulatory agency.
The posters will be made available on the Amicus website following their respective presentations at the congress.
For more information on the 26th International Annual Congress of the World Muscle Society, please visit www.wms2021.com.
About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company’s website at www.amicusrx.com, and follow us on Twitter and LinkedIn.