Clinical Trial Update: Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease [Phase 1, Phase 2]

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Summary

BMN701 was studied in late-onset Pompe disease in a Phase 1/2 trial focused on safety, tolerability, pharmacokinetics and pharmacodynamics. The study has comple…

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Diseases / indications

• Pompe Disease

Drugs mentioned

• BMN701

Snippet

Sponsor: BioMarin Pharmaceutical | Conditions: Pompe Disease | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2018-06-11