An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
- Status
- ENROLLING_BY_INVITATION
- Phase
- PHASE2
Clinical Trials Related to BioMarin Pharmaceutical
Related studies linked through entity extraction aliases.
Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
- Status
- NOT_YET_RECRUITING
- Phase
- PHASE4
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
- Status
- RECRUITING
- Phase
- PHASE3
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
- Status
- RECRUITING
- Phase
- PHASE2/PHASE3
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
- Status
- RECRUITING
- Phase
- PHASE2
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
- Status
- ENROLLING_BY_INVITATION
- Phase
- PHASE3
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE4
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE1
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
- Status
- RECRUITING
- Phase
- PHASE2
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
- Status
- RECRUITING
- Phase
- PHASE2
A Study to Assess Growth in Children With Idiopathic Short Stature
- Status
- RECRUITING
- Phase
- N/A
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
- Status
- RECRUITING
- Phase
- N/A
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
- Status
- RECRUITING
- Phase
- PHASE1/PHASE2
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
A Multicenter Multinational Observational Study of Children With Hypochondroplasia
- Status
- RECRUITING
- Phase
- N/A
VIrtual STudy in Achondroplasia for the US (VISTA)
- Status
- RECRUITING
- Phase
- N/A
A Study of BMN 255 in Participants With Non-Alcoholic Fatty Liver Disease And Hyperoxaluria
- Status
- WITHDRAWN
- Phase
- PHASE1
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
- Status
- RECRUITING
- Phase
- N/A
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
- Status
- TERMINATED
- Phase
- PHASE1