Drug Profile

Mucopolysaccharidosis I therapeutic - bluebird bio

Alternative Names: HSC LVV-gene-modified stem cells - bluebird bio

Latest Information Update: 28 Jun 2023

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

28 Jun 2023 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-I in USA (Parenteral)
10 May 2019 Preclinical trials in Mucopolysaccharidosis I in USA (Parenteral)

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