Odyssey Therapeutics and Sionna Therapeutics have filed IPO paperwork, swelling the pack of startups that will provide an ...

Vertex Pharmaceuticals (NASDAQ:VRTX – Get Free Report)‘s stock had its “overweight” rating restated by equities researchers ...

An additional 94 non-F508del CFTR mutations and a boxed warning on liver injury and liver failure were included in the updated prescribing information. 1507_1515del9, 2183A→G, A1067P ...

Image: Adobe Stock Keating and colleagues analyzed 398 patients (median age, 31 years; 59% male; 97% white) with cystic fibrosis aged at least 12 years who possessed the F508del-minimal function ...

It markets TRIKAFTA/KAFTRIO for people with CF with at least one F508del mutation for 2 years of age or older; SYMDEKO/SYMKEVI for people with CF for 6 years of age or older; ORKAMBI for CF ...

Under the deal, eligible patients – aged 12 or over who have two copies of the F508del mutation gene, or one copy of F508del and a minimal function (MF) mutation – will be able to get ...

If approved Symkevi will be used in combination with Kalydeco and be the treatment for the CFTR protein defect in CF patients who have one copy of the F508del mutation and a copy of one of 14 ...

in people 6 years and older who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to ALYFTREK. Vertex raised its CF patient estimates Zai Lab (ZLAB ...