Deputy Broughan welcomes today’s announcement that Spinraza is approved for children with spinal muscular atrophy (SMA) following a long and difficult campaign by families of children affected by this rare, muscle-wasting disease. In Ireland. there are around 70 people, 26 of whom are children, with SMA. It is the number one genetic cause of death for infants worldwide and will affect around 1 in 11,000 babies born worldwide each year. Spinraza or Nusinersen is produced by Biogen and has been the first treatment for Spinal Muscular Atrophy (SMA). It was available in most of the other European countries but not in Ireland.
Deputy Broughan recently submitted a Private Members’ Motion on Spinraza and it is now on the clár of the Dáil. The motion is supported by Deputies Thomas Pringle, Joan Collins, Clare Daly, Mick Wallace, Catherine Connolly, Maureen O’Sullivan, Gino Kenny, Bríd Smith, and Richard Boyd Barrett and Deputy Broughan hopes for an early opportunity to debate the motion.
There are approximately 70 people, including 26 children, in Ireland with Spinal Muscular Atrophy (SMA), which is a rare and life-limiting muscle-wasting condition. SMA is the number one genetic cause of death for infants worldwide and will affect around 1 in 11,000 babies born worldwide each year. Spinraza or Nusinersen, which is produced by Biogen, is the first treatment for Spinal Muscular Atrophy (SMA) and is available in 25 of the 28 countries in the European Union but in February 2019, the HSE did not approve Spinraza for reimbursement in Ireland and negotiations with Biogen are ongoing.
Last week, Deputy Broughan wrote about access to Spinraza for SMA sufferers. Another treatment for a rare disease that he has consistently been raising has been access to Kuvan for people with PKU (Phenylketonuria).
Today, Deputy Broughan received the following welcome response from the HSE following his most recent Parliamentary Question on the matter:
Yesterday was International Rare Disease Day, which has been celebrated in Ireland since 2008. Deputy Broughan consistently raises the issue of rare diseases and access to medicines and treatments for various rare diseases in Dáil Éireann.
According to the HSE website “A ‘rare disease’ is defined in Europe as a life-threatening or chronically debilitating disease affecting no more than 5 people per 10,000. There are an estimated 6-8,000 known rare diseases affecting up to 6% of the total EU population, (at least 30 million Europeans), and perhaps up to 300,000 Irish people during their lives.