Pompe disease

UK approval for Pompe Disease therapy

16 August 2023 | By

The MHRA has granted marketing authorisation for a two-component therapy for adults with late-onset Pompe Disease, a rare disorder.

news

CHMP meeting highlights – December 2022

19 December 2022 | By Catherine Eckford (European Pharmaceutical Review)

A total of five drugs were recommended for approval at the CHMP’s December meeting, including a gene therapy and two cancer drugs.

white pills spilling from a bottle forming the shape of a DNA double helix - idea of treatment for inherited/genetic disease

news

Nexviazyme^® improves respiration and mobility long-term in Pompe disease patients

10 February 2022 | By Hannah Balfour (European Pharmaceutical Review)

Two-year data shows Pompe disease patients treated with Nexviazyme® (avalglucosidase alfa) have sustained improvements in respiratory function and mobility.

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Recommended

Pompe disease

UK approval for Pompe Disease therapy

CHMP meeting highlights – December 2022

Nexviazyme® improves respiration and mobility long-term in Pompe disease patients

Nexviazyme^® improves respiration and mobility long-term in Pompe disease patients