Amicus Therapeutics’ treatment for a rare muscle disorder called Pompe disease was approved by the U.S. health regulator, the drugmaker said on Thursday, ending its years-long efforts to launch the therapy.
Tag Archive for: muscle disorder
Astellas Pharma will license and further develop a gene therapy from Kate Therapeutics aimed at addressing XLMTM amid safety concerns about its own experimental XLMTM treatment.
The European Medicines Agency’s decision comes amid delays for the drug in the United States.
