THE ONLY SMA GENE THERAPY IN HUMAN TRIALS
AVXS-101, the therapy evaluated in this study for SMA patients, has been granted Orphan Drug Designation for the treatment of all types of spinal muscular atrophy (SMA) and Priority Medicines (PRIME) designation by the EMA.
THE HEREDITARY NATURE OF SMA.
Inheriting a mutated or missing SMN1 gene prevents the body from adequately producing the SMN (survival motor neuron) protein—which is critical to the function of nerves that control our muscles—leading to the debilitating muscle weakness caused by SMA.
That’s why we need your family’s help with the STR1VE-EU research trial studying the effectiveness of gene therapy in the treatment of SMA. In this study, the gene therapy will be administered through an intravenous infusion (needle inserted into a vein); it will deliver a new gene that can produce the Survival Motor Neuron (SMN) protein that is missing in the spinal cord and motor nerves of children with SMA. By producing the SMN protein, the new gene may help improve a child’s SMA symptoms.