Sma: prima volta in Italia terapia genica a neonata – Sanità

By On Apr 27, 2022

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(ANSA) – BARI, 27 APR – E’ stata somministrata nell’ospedale
pediatrico Giovanni XXIII di Bari, per la prima volta in Italia,
la terapia genica ad una neonata di appena 23 giorni alla quale
è stata diagnosticata la Sma (atrofia muscolare spinale) due
settimane fa. La diagnosi è stata possibile poiché la Puglia,
unica regione italiana, ha reso obbligatorio lo screening per
tutti i neonati. Alla bimba è stato somministrato il farmaco
Zolgensma.

“È il miracolo della diagnosi precoce della Sma”, commenta il
presidente della Commissione regionale Bilancio e programmazione
Fabiano Amati, che ha proposto la legge per introdurre l’obbligo
di screening su tutti i neonati. “Per la prima volta –
sottolinea – la terapia genica a un neonato senza sintomi con
età inferiore a un mese. In questo stato di malattia,
diagnosticato attraverso lo screening obbligatorio e solo dopo
sette giorni dalla nascita, possiamo coltivare la speranza di
risultati inimmaginabili”. “Da questo momento – continua – mi
batterò affinché lo screening obbligatorio non sia più solo un
beneficio dei neonati pugliesi ma di tutti i bimbi italiani”.

“Siamo l’unica Regione in Italia ad aver stabilito per legge
lo screening obbligatorio della Sma – spiega il governatore
pugliese Michele Emiliano – questo ha consentito alcuni giorni
fa di individuare e oggi di effettuare la prima somministrazione
della terapia genica su una neonata che non ha sviluppato ancora
alcun sintomo della malattia neurodegenerativa. Grazie ai
professionisti della sanità pugliese che offrono una prova di
grande efficienza e al Consiglio regionale che ha dimostrato
grande sensibilità e lungimiranza con l’approvazione della
legge”. (ANSA).

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