The Pompe community has been closely involved with the development of this novel therapy of ERT taken together with a pharmacological Chaperone, and it has finally been approved in the UK for Late-onset Pompe disease. Now known by the name Pombiliti + Opfolda, it has lately been available in the UK through an early access to medicines scheme (EAMS), but now has been given the NICE seal of approval. To patients, the main difference will be that homecare should again allow full or partial independence from the homecare nursing service, either by self-infusion, or by the nurse leaving shortly after canulating.
You can read the full press release from Amicus here:
Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the United Kingdom