VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pomp ...
A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All Patients A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacoki ...
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 in Ambulatory and Ventilator-free Patients with Pompe Disease A Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficac ...
A Three-month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 versus Myozyme®/Lumizyme® in Patients with Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment with VAL-1221 in all Patients A Three-month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacoki ...
Late-Onset GSD-II (Pompe Disease) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10045253;Term: Type II glycogen storage disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Late-Onset GSD-II (Pompe Disease) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: ...
Product Name: VAL-1221 Product Code: VAL-1221 INN or Proposed INN: VAL-1221 Trade Name: Myozyme Product Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA Product Name: VAL-1221 Product Code: VAL-1221 INN or Proposed INN: VAL-1221 Trade Name: Myozyme Product ...
A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for P ...
A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and ...