BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

Fabry Disease Clinical Trial Pipeline Major pharmaceutical companies are working to advance the pipeline space and unlock the future growth ...

Also Read: Sanofi Genzyme Facility Draws FDA Criticism Over Alleged Quality Lapses An independent data monitoring committee ...

Orchard Therapeutics has tasked Robin Kenselaar with accelerating growth of Lenmeldy, the world’s most expensive drug, ...

The recent FDA approvals of two treatments for molluscum contagiosum may represent a shift in how clinicians view this disease, according to a speaker at Masters of Pediatric Dermatology.In an ...

After winning approval in the United States back in September, Sanofi Genzyme’s multiple sclerosis drug, aubagio, has won approval for use in Australia. The Australian Therapeutic Goods ...

Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) in ...

Information presented by Genzyme about Shire’s drug was inaccurate and inconsistent with the drug’s Summary of Product Characteristics, was misleading and unsubstantiated and disparaged a ...

Drug addiction is a condition in which there is uncontrolled use of a substance with a harmful outcome. Drugs overstimulate the brain’s reward circuit, producing pleasure or euphoria. With repeated ...

Ozempic, the blockbuster GLP-1 drug that was originally approved to treat type 2 diabetes, has now also been approved by the US Food and Drug Administration to reduce certain risks associated with ...

An audit released by the Inspector General of the U.S. Department of Health and Human Services, the parent organization of the Food and Drug Administration (FDA), is not a pleasant read. Let us turn ...