Amicus Therapeutics could be just weeks away from getting EU approval for its new Pompe disease therapy Pombiliti after getting a positive opinion on the drug from the EMA's human medicines
Amicus Therapeutics’ first oral drug for the rare condition Fabry disease has been approved by the FDA, giving patients an alternative to conventional injected enzyme replacement therapy.
In an industry growing as quickly as cell therapy, keeping up with the demands of manufacturing and treatment is a challenge for the whole industry to tackle together, including stakeholder
Drug discovery is usually a goal-driven and iterative process, as researchers look for innovative new routes to target particular diseases and study those diseases in afflicted patients to learn ho