Pompe disease
Sanofi extends its Pompe range with first Nexviazyme OK
Phil Taylor
fda, Nexviazyme, Pompe disease, rare disease, regulatory approval, Sanofi, Sanofi Genzyme
0 Comment
Boost for AZ as Ultomiris gets CHMP nod for expanded label
Phil Taylor
Alexion, AstraZeneca, Kyowa Kirin, Nexviadyme, Nouryant, Parkinson's disease, Pompe disease, rare disease, Sanofi Genzyme, Soliris, Ultomiris
0 Comment
FDA to expedite development of two Sanofi rare disease drugs
Richard Staines
fda, immune thrombocytopenia, Pompe disease, R&D, Rare diseases, Sanofi
0 Comment
Healthcare/ News/ US
Thanks Mr President, but you’re wrong about FDA, says rare disease group
Andrew McConaghie
Donald Trump, fda, NORD, orphan drugs, Pompe disease, Rare diseases
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NORD patient advocacy group responds to Congress speech