This is a single arm, open-label continued access protocol of drisapersen for the treatment of male subjects with Duchenne muscular dystrophy (DMD) having dystrophin mutations correctable by drisapersen-induced DMD Exon 51 skipping. The purpose of this continued access protocol is to offer pre-approval access to drisapersen for the treatment of subjects with DMD who previously participated in eligible drisapersen studies. The protocol will collect safety data required to assure subject safety and periodic efficacy data on muscle function.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Drisapersen will be supplied as 3 millilitre (mL) vials containing 1mL sterile solution for subcutaneous injection. The strength of drisapersen solution will be 200 mg/mL.
Safety as assessed by the collection of adverse events (AEs)
AEs will be collected from the start of Study Treatment and until 5 days post last-dose (at follow up).
Time frame: Baseline to Week 48
Safety as assessed by laboratory parameters
Absolute values and changes over time of hematology, clinical chemistry, and urinalysis
Time frame: Baseline to Week 48
Safety as assesses by electrocardiogram (ECG) intervals
Time frame: Baseline to Week 48
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