Catalyst Pharmaceuticals, Inc. — Biomedical Watch V2

Traitements4programmes

Essais2liés

Publications3liées

SourceDBlocale

Traitements

4

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
Amifampridine PhosphateEfficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.	Myasthénie	Phase 3	À vérifier	United States	À vérifier
VamoroloneThe goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).	Myopathies	À vérifier	À vérifier	United States, Puerto Rico	À vérifier
Amifampridine PhosphateEfficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.	Myasthénie	Phase 3	À vérifier	United States	À vérifier
VamoroloneThe goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).	Myopathies	À vérifier	À vérifier	United States, Puerto Rico	À vérifier

Essais cliniques

2

Molécule	Indication / population	Phase	NCT	Titre	Statut
Vamorolone	Myopathies	À vérifier	NCT06564974	DMD-001 SUMMIT — Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT	RECRUITING
Amifampridine Phosphate	Myasthénie	Phase 3	NCT03304054	Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG	COMPLETED

Publications

3

Molécule	Indication / population	Titre	Journal	Date
Vamorolone		Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial.	Neurology
Vamorolone		Prednisone, not vamorolone, suppresses novel serum bone and cartilage biomarkers associated with growth failure in children with Duchenne muscular dystrophy.	Scientific reports
Amifampridine Phosphate		Long-term Efficacy and Safety of Amifampridine Phosphate (Firdapse) in Japanese Patients with Lambert-Eaton Myasthenic Syndrome (LMS-005 Study).	Internal medicine (Tokyo, Japan)