Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

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The Amicus acquisition will bolster BioMarin’s rare disease portfolio, while potentially supporting the company’s revenue ...

BioMarin said Friday it will acquire Amicus Therapeutics for $4.8 billion in a deal that adds two rare disease drugs to its ...

BioMarin Pharmaceutical is building up its pipeline with the acquisition of Inozyme Pharma in a $270 million deal centered on an enzyme replacement therapy that could become the first FDA-approved ...

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 ...

Breakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE) U.S. FDA Confirmed ...

A couple struggling to access a vital cancer treatment say they are "extremely stressed" by the uncertainty in its supply.

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 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 Indian Medical Parliamentarians Forum has urged urgent government action to prevent life-threatening treatment ...

"There are three digestive enzymes made by your pancreas," says Mary Vouyiouklis Kellis, MD, a specialist in the department of endocrinology, diabetes and metabolism at the Cleveland Clinic. "Most ...