Spinal Muscular Atrophy: Maximizing Quality of Life

Spinal Muscular Atrophy: Maximizing Quality of Life

Children with Spinal Muscular Atrophy (SMA) have complex medical needs which can lead to diminished quality of life.1 Historically, patients diagnosed with SMA and their families report significant stress and a low quality of life.2-4 It is incorrect to assume that...
Disease-Modifying Therapies for Treating SMA

Disease-Modifying Therapies for Treating SMA

Until the approval of nusinersen (Spinraza), treatment for spinal muscular atrophy was largely supportive. While patients with SMA enjoy benefits from respiratory therapy, physical therapy, nutrition support, etc., these therapies are aimed at maximizing residual...