It’s a big day for Sanofi as the pharmaceutical giant welcomes two European drug approvals, both for rare enzyme deficiencies. One regulatory nod ushers in the first treatment in Europe for a ...
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BioMarin Beats on Q3 Earnings, Seeks to Divest Hemophilia Gene Therapy
BioMarin Pharmaceutical BMRN reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks ...
Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first test ...
When CEO Paul Hudson took over at Sanofi in 2019, he launched a “play to win” strategy centered on first-in-class and best-in-class drugs in therapeutic areas where the company was lacking. On Tuesday ...
In early 2022, Connor started a new medication called Nexviazyme, but each treatment took 13 hours. This summer, he received ...
MPS II, also called Hunter syndrome, is a rare genetic disease that affects over 2,000 individuals, primarily males, world-wide, and leads to behavioral, cognitive, and physical symptoms ultimately ...
FDA approves avalglucosidase alfa-ngpt (Nexviazyme) for the treatment of patients 1 year and older with late-onset Pompe disease. FDA announced yesterday the approval of avalglucosidase alfa-ngpt, ...
In early trial data, INZ-701 was associated with several clinical benefits including a reduction or stabilization of carotid intima-media thickness. The Food and Drug Administration (FDA) has granted ...
The US Food and Drug Administration (FDA) has approved a new enzyme replacement therapy, avalglucosidase alfa (Nexviazyme, Genzyme), for patients aged 1 year and older with late-onset Pompe disease.
In a recent study posted to the medRxiv* preprint server, researchers evaluated the impact of the coronavirus disease 2019 (COVID-19) pandemic on the rates of pancreatic enzyme replacement therapy ...
Pancreatic enzyme replacement therapy (PERT) can help improve or resolve symptoms of exocrine pancreatic insufficiency (EPI). If symptoms persist, a person may consider speaking with a doctor about ...
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