What is Spinal Muscular Atrophy

Spinal muscular atrophy, also known as SMA, is a hereditary neuromuscular disease of childhood and youth characterized by the progressive death of motor neurons , the nerve cells of the spinal cord  that give the  muscles  the command of movement.

The severity of this disabling disease is established by a precise clinical picture which divides them into three types and which classifies them starting from a progressive loss of muscle control, atrophy and physical weakness.

New drug for spinal muscular atrophy 

It is called Nusinersen therapy and it is, as well explained by Notiziariochimicofarmaceutico.it , a “gene” drug that would be able to activate the function of the SMN2 gene, complementary to the inactive one of the disease. The drug, injected through the lumbar, spreads throughout the body, reaching the degenerated motor neurons and effectively relieving the symptoms of SMA.

The new drug underlying this therapy was approved by the Italian Medicines Agency (AIFA) in September 2017.

The European Commission then issued the AIC valid throughout the European Union on May 30 of the same year.

Where to find the new drug for muscle atrophy

Nusinersen therapy is already prescribed in 18 authorized and operational Italian centers located in eleven regions: Campania, Emilia Romagna, Friuli Venezia Giulia, Lazio, Liguria, Lombardy, Marche, Piedmont, Sicily, Tuscany and Veneto, the detailed map of the places where this therapy is in progress and can be consulted on the FamiglieSMA website at the dedicated page. 

New structures will be added to these structures, with the aim of creating networks for sharing and training medical personnel capable of administering the new therapy. 

Consult the web: useful pages to stay updated on the subject are the Observatory for rare diseases  and Famigliesma.org

Here is the ENDEAR study published by ClibnicalTrials.org, US National Library of Medicine, aimed at proving the efficacy of this therapy.