2024-04-23 12:05:38
(ANSA) – ROME, APRIL 23 – Children with type 2 spinal muscular atrophy, with symptoms that appear between 6 and 18 months of age, will also be able to benefit from treatment with Onasemnogene abeparvovec (Zolgensma©), thanks to the expansion of reimbursement criteria for gene therapy by the Italian Medicines Agency (AIFA). This therapy will allow us to directly address the genetic cause of SMA by replacing the function of the missing or non-functioning SMN1 gene in order to halt the progression of the disease with a single intravenous administration. “Early administration of this treatment allows for better results to be achieved in halting the progression of the disease. Prenatal diagnosis plays an important part – explains Marika Pane, Clinical Director of the Nemo Pediatric Center in Rome and Associate Professor of Child Neuropsychiatry at the Catholic University of Sacred Heart of Rome – It must be taken into account that the degeneration of motor neurons begins before birth, intensifies rapidly and that the innovative aspect of this treatment is that it intervenes directly on the genetic defect with a single administration; therefore, it is carried out only once in a lifetime”. SMA, characterized by the progressive loss of motor skills, is a “disease continuum”, which affects approximately 40-50 children in Italy every year, with a range of symptoms that can vary in severity. “We welcome the news – declares Anita Pallara, president of the SMA Families Association -. We know that gene therapy increases its effectiveness and, therefore, improves the patient’s response the sooner it is administered. For this reason, we reiterate the importance of neonatal screening, which must be extended to all regions in order to guarantee the same right to health throughout the national territory and the necessary accompaniment in the subsequent post-treatment follow-up phases, through care by specialized centers”. Supporting the reimbursement extension was the completion of the phase III SPR1NT study, which demonstrated how small patients with three copies of the SMN2 backup gene, treated pre-symptomatically, achieved motor goals appropriate for their age , including the ability to stand and walk. (HANDLE).
2024-04-23 12:05:38