Ana de Barros, PhD, managing science editor —

Amicus Therapeutics announced the beginning of the availability of Galafold (migalastat) to treat Fabry disease in Japan, making this the first oral precision medicine available for patients with an amenable mutation in that country. Fabry disease is caused by mutations in the GLA gene, which lead to a defective…

April is Fabry Disease Awareness Month, and Amicus Therapeutics helped to raise awareness about the disease, as well as Pompe disease, by participating in a series of activities throughout the month. Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha…

Amicus Therapeutics has launched Healing Beyond Disease, an initiative designed to celebrate people living with rare diseases, like Fabry disease, and their loved ones. “We are thrilled and honored to officially launch Healing Beyond Disease, our unique promise to further serve the needs of the rare disease community in extraordinary…

Biotechnology companies Shire and NanoMedSyn are collaborating on research to evaluate a potential enzyme replacement therapy (ERT) for lysosomal storage disorders, including Fabry disease. Under the terms of the new agreement, the two companies will perform preclinical evaluations on NanoMedSyn’s proprietary synthetic derivatives, called AMFA, in combination with recombinant…

The U.S. Food and Drug Administration will be giving priority review to Amicus Therapeutics’ Fabry disease therapy Galafold (migalastat). Fabry disease is caused by mutations of the GLA gene. Amicus asked the FDA to approve Galafold for Fabry patients older than 16 with mutations that can respond to migalastat. Up to half…

Amicus Therapeutics recently announced the launch of Galafold (migalastat) in Spain for the long-term treatment of anyone who has Fabry disease and is older than 16 years. Following final regulatory adjustments, Galafold is now reimbursed in Spain for age-eligible patients with a confirmed diagnosis of Fabry disease and who have…

The Canadian Drug Expert Committee (CDEC) has recommended in favor or reimbursement of Galafold (migalastat) for long-term treatment of adults with a confirmed diagnosis of Fabry disease and who have an amenable mutation, Amicus Therapeutics announced. CDEC will now start conversations with regulatory authorities to make Galafold available to Canadian…