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Results For "rare-disease"

234 News Found

FDA approves Nexviazyme for late-onset Pompe disease
Drug Approval | August 09, 2021

FDA approves Nexviazyme for late-onset Pompe disease

Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells


Sanofi to acquire Translate Bio
Biotech | August 07, 2021

Sanofi to acquire Translate Bio

Advances deployment of mRNA technology across vaccines and therapeutics development


Lonza announces expansion plans for mammalian manufacturing facilities
News | May 07, 2021

Lonza announces expansion plans for mammalian manufacturing facilities

The facility is expected to be completed in 2024


Dr. Reddy's Laboratories launches Sapropterin Dihydrochloride powder for oral solution in US
News | April 08, 2021

Dr. Reddy's Laboratories launches Sapropterin Dihydrochloride powder for oral solution in US

Sapropterin dihydrochloride powder for oral solution is available in 100 mg unit dose