“OneGene”, a patient support program promoted by Novartis Gene Therapies, to support children living with spinal muscular atrophy (SMA) treated with gene therapy and their families is launched. The Program was created precisely to accompany the treatment path and the subsequent follow-up phases after treatment with Zolgensma *, the first and only gene therapy for SMA that is administered only once in a lifetime and intervenes on the genetic cause of the disease, and it does so through a series of services that can be activated on the basis of the specific needs encountered by patients and families.
Considered the leading genetic cause of infant death, SMA is a rare genetic neuromuscular disease caused by the lack of a functioning Smn1 gene, resulting in a rapid and irreversible loss of motor neurons, which impairs muscle function.
Through ‘OneGene’ it will be possible to carry out blood sampling at the patient’s home and delivery to the analysis laboratory, with the possibility for the specialist doctor to receive and consult the related reports, and the organization and logistic management of transfers from home to the specialized clinical center (and vice versa) during check-ups. Specialist visits during the follow-up phase after the administration of gene therapy will be essential in order to constantly follow the developments in the patient’s state of health: for this reason the program also provides for the organization and provision of specialist visits, meetings with a nutritionist to educate and support families in the management of dysphagia, or difficulties in swallowing, physiotherapy consultations at the patient’s home to help families in patient mobility.
“The project wants to give an important message to families who access new pharmacological treatments, last of all gene therapy – comments Valeria Sansone, clinical-scientific director of the NemO Clinical Center in Milan – It is fundamental and necessary, in fact, that children continue to be monitored from a clinical and functional point of view, with periodic and targeted follow-ups. Because maintaining a correct path of care – he underlines – means evaluating changes over time and in an integrated way, following the growth path of the little ones according to recognized standards of care “.
“This is an important historical moment for all patients and families living with SMA: today the attention and awareness that it is a complex disease that requires early diagnosis and timely treatment are increasingly widespread also in the general population – says Anita Pallara, president of SMA Families – post-treatment support programs with gene therapy respond to the needs and requirements of the SMA world, which as an association representing patients we have welcomed with great joy and enthusiasm “.
All information is available on the platform www.signsofsma.it, promoted with the unconditional contribution of Novartis Gene Therapies and with the patronage of the Italian Federation of pediatricians (Fimp), the Nemo Clinical Centers and the association of patients Families Sma. A point of reference for those who face the complexity of this pathology on a daily basis.
“Our mission is to bring innovation and be concretely alongside patients and their families, not only from a therapeutic point of view, but also through targeted support programs – says Filippo Giordano, general manager of Novartis Gene Therapies – The Program support to patients and their families “OneGene” and the site www.signofsma.it in this sense they represent two significantly tangible examples of how the new way of interpreting medicine must be increasingly transversal and at the same time focused on the needs of those who every day have to deal with an extremely complex pathology such as Sma. Like Novartis, we have been involved in this area for years and will continue to work precisely in this direction in concert with the scientific community and with the patient association. “