The disease must be type 2, late-onset, in people between the ages of 2 and 18 who have had no prior treatment and who can sit but not walk, the health agency said.
SMA is a genetic condition linked to an alteration in chromosome 5, specifically the gene responsible for producing the SMN protein, which is essential for the survival of specialized nerve cells called motor neurons, or motor neurons.
Various high-tech treatment strategies for this type of disease have appeared in recent years, giving patients hope of treatment.
For SMA, preliminary studies have shown that gene therapy with the drug approved for this study increases levels of the SMN protein and improves motor neuron function in patients.
This is the first clinical trial of gene therapy to be carried out in this South American country, thanks to a joint work between Invima, the Colombian Medical Genetics Association, and faculty members of the Institute of Inherited Metabolic Disorders of the Pontificia Universidad Javeriana.
After the evaluation process to establish a favorable risk-benefit ratio, the development of this medical research will take place at the San Ignacio University Hospital in Bogotá.
ro/otf