BioMarin holds the line on bleeds with 4-year valrox update on hemophilia A — but what's this about another decline in Factor 8 levels?

John Carroll

Editor & Founder

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a …

Sign up to read this article for free.

Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.

BECOME A PREMIUM SUBSCRIBER

BioMarin holds the line on bleeds with 4-year valrox update on hemophilia A — but what's this about another decline in Factor 8 levels?

John Carroll

Editor & Founder

Sign up to read this article for free.

Applied Therapeutics' rare disease drug gets a CRL

FDA is looking into hematology risks after patients take Bluebird's Skysona gene therapy

PTC reports Phase 2 ALS fail, sells priority review voucher for $150M

Bristol Myers sues the federal government over 340B, following other drugmakers

Norbert Bischofberger's time as Kronos CEO ends as biotech seeks new life

Novartis to cut 139 jobs in NJ as part of commercial refocus

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

John Carroll

Editor & Founder

Sign up to read this article for free.

more like this

Ap­plied Ther­a­peu­tic­s' rare dis­ease drug gets a CRL

FDA is look­ing in­to hema­tol­ogy risks af­ter pa­tients take Blue­bird's Skysona gene ther­a­py

PTC re­ports Phase 2 ALS fail, sells pri­or­i­ty re­view vouch­er for $150M

Bris­tol My­ers sues the fed­er­al gov­ern­ment over 340B, fol­low­ing oth­er drug­mak­ers

Nor­bert Bischof­berg­er's time as Kro­nos CEO ends as biotech seeks new life

No­var­tis to cut 139 jobs in NJ as part of com­mer­cial re­fo­cus

BioMarin holds the line on bleeds with 4-year valrox update on hemophilia A — but what's this about another decline in Factor 8 levels?

Applied Therapeutics' rare disease drug gets a CRL

FDA is looking into hematology risks after patients take Bluebird's Skysona gene therapy

PTC reports Phase 2 ALS fail, sells priority review voucher for $150M

Bristol Myers sues the federal government over 340B, following other drugmakers

Norbert Bischofberger's time as Kronos CEO ends as biotech seeks new life

Novartis to cut 139 jobs in NJ as part of commercial refocus