BioMarin Pharmaceutical — Biomedical Watch V2

Traitements9programmes

Essais4liés

Publications6liées

SourceDBlocale

Traitements

9

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
BMN 044 IV 6 mg/kgThe aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period.	Myopathies	Phase 2	À vérifier	Belgium, Italy, Netherlands, Sweden	À vérifier
BMN 044 IV 9 mg/kgThe aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period.	Myopathies	Phase 2	À vérifier	Belgium, Italy, Netherlands, Sweden	À vérifier
BMN 044 SC 6 mg/kgThe aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period.	Myopathies	Phase 2	À vérifier	Belgium, Italy, Netherlands, Sweden	À vérifier
BMN 351This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the 351-201 study.	Myopathies	Phase 1/2	À vérifier	Italy, Netherlands, Spain, Turkey (Türkiye), United Kingdom	À vérifier
DrisapersenThis is a phase IIIb, multi-centre, open-label extension study in male subjects with DMD who previously have been treated with drisapersen, aiming at assessing the safety and efficacy of drisapersen.	Myopathies	À vérifier	À vérifier	United States, Argentina, Australia, Belgium, Bulgaria, Czechia, France, Germany, …	À vérifier
BMN 044 IV 6 mg/kgThe aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a longer treatment period.	Myopathies	Phase 2	À vérifier	Belgium, Italy, Netherlands, Sweden	À vérifier
DrisapersenThis is a phase IIIb, multi-centre, open-label extension study in male subjects with DMD who previously have been treated with drisapersen, aiming at assessing the safety and efficacy of drisapersen.	Myopathies	À vérifier	À vérifier	United States, Argentina, Australia, Belgium, Bulgaria, Czechia, France, Germany, Israel, Italy, Japan, Netherlands, Norway, Poland, Russia, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom	À vérifier
BMN 351This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the 351-201 study.	Myopathies	Phase 2	À vérifier	Italy, Netherlands, Spain, Turkey (Türkiye), United Kingdom	À vérifier
BMN 351This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the 351-201 study.	Myopathies	Phase 1/2	À vérifier	Italy, Netherlands, Spain, Turkey (Türkiye), United Kingdom	À vérifier

Essais cliniques

4

Molécule	Indication / population	Phase	NCT	Titre	Statut
BMN 351	Myopathies	Phase 1/2	NCT06280209	A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy	ACTIVE_NOT_RECRUITING
BMN 351	Myopathies	Phase 2	NCT07573631	An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy	ENROLLING_BY_INVITATION
Drisapersen	Myopathies	À vérifier	NCT02636686	Extension Study of Drisapersen in DMD Subjects	NO_LONGER_AVAILABLE
BMN 044 IV 6 mg/kg	Myopathies	Phase 2	NCT02958202	Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)	TERMINATED

Publications

6

Molécule	Indication / population	Titre	Journal	Date
Drisapersen		A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy.	Neuromuscular disorders : NMD
Drisapersen		Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy.	Annals of clinical and translational neurology
BMN 351		BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal and Cardiac Muscle Lead to Preservation of Motor Function in a Mouse Model of Exon 51 Skip-Amenable Duchenne Muscular Dystrophy.	Nucleic acid therapeutics
BMN 351		Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy.	Nucleic acid therapeutics
Drisapersen		Characteristics of Patients Receiving Novel Muscular Dystrophy Drugs in Trials vs Routine Care.	JAMA network open
Drisapersen		Spending on Targeted Therapies for Duchenne Muscular Dystrophy.	JAMA