Important Safety Information Indication Elfabrio ® (pegunigalsidase alfa for injection) is an enzyme replacement therapy indicated for the longterm treatment of adult patients with a confirmed ...

Health Canada has approved a new drug treatment for adults living with Fabry disease, a rare genetic disorder that can affect multiple organs and impact the quality of life.

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 ...

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 ...

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.

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 ...

The FDA has placed a hold on Denali Therapeutics’ plans to launch a phase 1 rare disease trial, citing concerns about immune ...

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

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 ...

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 ...

"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 ...