Drug Profile

Research programme: enzyme replacement therapeutics - SylamoreBio

Latest Information Update: 22 Apr 2026

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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 Yes

Preclinical Mucopolysaccharidosis I
Research Fabry's disease; Globoid cell leukodystrophy; Glycogen storage disease type II; GM1 gangliosidosis; Mucopolysaccharidosis III; Mucopolysaccharidosis IV
Discontinued Gaucher's disease

16 Apr 2026 Early research in Mucopolysaccharidosis III is ongoing in USA (Parenteral) (SylamoreBio pipeline, April 2026)
16 Apr 2026 Discontinued for Gaucher's disease in USA (Parenteral) before April 2026 (SylamoreBio pipeline, April 2026)
16 Apr 2026 Early research in Fabry's disease in USA (Parenteral) before April 2026 (SylamoreBio pipeline, April 2026)

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