Italy : a newborn baby receives Zolgesma, an innovative drug against Sma (spinal muscular atrophy)
Sma (spinal muscular atrophy): the little patient suffering from Sma (spinal muscular atrophy), who was given the innovative drug Zolgesma for the first time in Puglia on 12 January at the Giovanni XXIII paediatric hospital of the Policlinico hospital-university of Bari, is doing well but is still under close clinical monitoring.
Sma (spinal muscular atrophy), Zolgesma infused to Apulian baby
This is the first child from Puglia, the third in Italy and the youngest ever to have undergone the treatment, which took place at the age of 3 months, who has had access to gene therapy.
This is an innovative drug, an example of sophisticated genetic engineering applied in the medical field for the treatment of a disease that, before the recent advent of new therapeutic strategies, had a consistently lethal outcome within two years of life,” explain paediatric hospital neurologists, Delio Gagliardi and Pasquale Conti.
Gene therapy represents a treatment opportunity that acts on the correction of the gene defect in a single administration over a lifetime, in addition to the other therapeutic options in use today, and is included in the list of medicines that can be paid for by the national health service for the treatment within the first six months of life of patients with a genetic diagnosis or clinical diagnosis of spinal muscular atrophy type 1″.
To undergo the treatment, the newborn child underwent preliminary tests in a Rotterdam laboratory that collects blood samples from all over Europe.
In the meantime, the health workers have participated in training by the pharmaceutical company.
The drug, customized to the weight of the little patient, was ordered from the hospital pharmacy and within a few days was shipped from a European distribution center in Ireland and stored in the pharmacy at a temperature of -70 degrees.
The baby was then infused with the drug and is now under close clinical monitoring.