Nusinersen (Spinraza) Announcement
SMA Australia welcomes the announcement by the Pharmaceutical Benefits Advisory Committee (PBAC) to include the treatment of pre-symptomatic patients to the PBS.
The pre-symptomatic window may be brief (weeks or several months), but we are excited that this is another step in the timely diagnosis and treatment for those living with Spinal Muscular Atrophy. This brand new generation of SMA will give families much greater hope for their child living with the condition.
The important step forward now is to continue to advocate for a national newborn screening program. Newborn Screening (NBS) may avoid the diagnostic odyssey that families commonly encounter and provide an opportunity to reduce morbidity resulting from a delayed diagnosis of SMA. The introduction of a pilot Newborn Screening Program in NSW recently to pick up new cases, will be a testament to the SMA community, and those families who should be able to have access to treatment upon diagnosis, rather than waiting for symptoms to occur.