DMD: pharmacological trials-2

  •  SRP4053 : A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
  • Essence : Study of SRP-4045 and SRP-4053 in DMD Patients
  • SRP 4658-102: An open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with DMD Amenable to Exon 51 Skipping

 

Acronym

SRP4053

Essence

SRP 4658-102

Study type BMR BMR BMR
Trial type Multicentre trial International multicentre trial International multicentre trial
Intervention SRP-4053 SRP-4053 / SRP-4045 Eteplirsen
Principal investigator
Laurent Servais Laurent Servais Laurent Servais
Sponsor SAREPTA SAREPTA SAREPTA
Fundings SAREPTA / FP7 SAREPTA / FP7 SAREPTA
Study status Ongoing Ongoing Ongoing
Recruitment status Closed Ongoing Ongoing
Population Child Child Child
Contact l.servais@institut-myologie.org l.servais@institut-myologie.org l.servais@institut-myologie.org
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