August 13, 2019 – Onasemnogene abeparvovec-xioi (Zolgensma) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 …

Onasemnogene abeparvovec-xioi (Zolgensma): Approval strategy with manipulated data? Read more »

May 29, 2019 – Spinal muscular atrophy (SMA) is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting. The severity of symptoms and age of onset varies by the type. Some types are apparent at …

Gene therapy: Zolgensma to treat spinal muscular atrophy Read more »

November 12, 2017 – Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor neuron disease with an onset during infancy that results in failure to achieve motor milestones and in death or the need for mechanical ventilation by …

Spinal Muscular Atrophy: Single-Dose Gene-Replacement Therapy Read more »