Pharmaceutical composition for treating, preventing or ameliorating spinal muscular atrophy and administration method thereof

Abstract

The present disclosure relates to a pharmaceutical composition for treating, preventing and/or ameliorating spinal muscular atrophy (SMA) and a method of administration thereof. A pharmaceutical composition for treating, preventing and/or ameliorating SMA according to an embodiment of the present disclosure may comprise a SMN protein, which is the cause of the onset of SMA. The pharmaceutical composition may be administered directly to a subject suffering from SMA via intracerebroventricular, intrathecal or intra-cisterna magna administration, thereby treating or preventing SMA or ameliorating symptoms of SMA. The pharmaceutical composition for treating, preventing or ameliorating SMA according to one embodiment of the present disclosure is safe, cost-effective and/or accessible compared to other previously approved drugs and can achieve substantially equivalent therapeutic effects.

Dependencies are inferred from the claim text.