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A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age, treatment naive, sitting, and never ambulatory

A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age, treatment naive, sitting, and never ambulatory

icon 2022-11-22
عنوان الدراسة
A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age, treatment naive, sitting, and never ambulatory
داعم الدراسة
Novartis Saudi Limited
دواء الدراسة
OAV101 (formerly AVXS-101)
رقم بروتوكول الدراسة
COAV101B12301
حالة الدراسة
Ongoing
موقع الدراسة
King Faisal Specialist Hospital and Research Center (Riyadh),King Fahad Specialist Hospital (Dammam)
مرحلة الدراسة
3