Amicus CEO 'proud of relentless commitment' to make Pompe disease treatment available 17-Aug-2023 By Liza Laws The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorizations for Pombliti + Opfolda, a two-component therapy to treat Pompe disease.
Paragon contracted to support Amicus’ preclinical gene therapy program 04-Jul-2019 By Maggie Lynch Amicus and Paragon enter a preclinical manufacturing agreement for the former’s lysomal disorder programs currently in development at Penn.
Amicus picks Brammer for preclinical, clinical gene therapy work 02-Jul-2019 By Vassia Barba Rare disease drug developer to work with Brammer Bio – which Thermo Fisher acquired in March – on gene therapy programs against Batten disease.