Pompe disease Archives -

With the safety of gene therapies thrown into the spotlight by a series of FDA clinical holds, Astellas

Astellas says Pompe gene therapy clears safety hurdle

https://pharmaphorum.com/news/astellas-says-pompe-gene-therapy-clears-safety-hurdle/

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its

Sanofi extends its Pompe range with first Nexviazyme OK

https://pharmaphorum.com/news/sanofi-extends-its-pompe-range-with-first-nexviazyme-ok/

Fresh from its takeover of Alexion, AstraZeneca has picked up a recommendation in the EU for an expansion

Boost for AZ as Ultomiris gets CHMP nod for expanded label

https://pharmaphorum.com/news/boost-for-az-as-ultomiris-gets-chmp-nod-for-expanded-label/

The FDA has agreed to expedite development of two rare disease drugs from Sanofi. The most significant of

FDA to expedite development of two Sanofi rare disease drugs

https://pharmaphorum.com/news/fda-to-expedite-development-of-two-sanofi-rare-disease/

NORD patient advocacy group responds to Congress speech

Thanks Mr President, but you’re wrong about FDA, says rare disease group

https://pharmaphorum.com/news/thanks-mr-president-youre-wrong-fda-says-rare-disease-group/

Pompe disease