Make rare disease treatment SPINRAZA,for SPINAL MUSCULAR ATROPHY available in SOUTH AFRICA

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Why is SPINRAZA not available in South Africa?

In December 2016, pharmaceutical giants Biogen announced that Nusinursen, an antisense oligonucleotide also known as Spinraza received FDA approval for all types of Spinal Muscular Atrophy. Spinraza,  labelled as a miracle drug assists in muscle development by targeting the SMN2 GENE and reverses symptoms in SMA patients. Generally, with SMA, the younger the individual is diagnosed with SMA the more severe is the condition, yet infants have shown great benefits from commencing the treatment as early as possible. Older individuals are also showing promising results.

Surprisingly this drug is currently only available in America,Canada and some parts of Europe. Biogen has also applied for it to be available in Japan and Australia.  South Africa has been completely ignored even though there are many who could benefit from this life saving treatment. 

What is SMA?

 Spinal Muscular Atrophy is an autosomal recessive degenerative genetic disorder and is considered the #1 genetic cause of infant death. SMA is caused because of a mutation in the SMN1 gene, which is the gene responsible for producing protein needed for muscle development. In individuals with SMA, the smn1 gene is either damaged or completely absent.  As a result, too little of this vital protein is produced and survival motor neurons die leading to muscle atrophy or wastage. Although there is a missing gene in most cases, a backup gene known as the SMN2 GENE which is a non-fully functional copy of the SMN1 gene exists in humans.

There are 4-5 different forms of SMA, varying according to severity of the condition, with type 0-1 being the most severe. Since SMA is a degenerative condition, muscle wastage occurs with time, disabling an individual and making them susceptible to respiratory infections. In some of the more severer forms, the ability to swallow, talk and even to smile is lost. A simple cold or flu if not treated in time and properly can result in death.

Parents in America are winning lawsuits against medical insurance companies even though the price tag of this drug is shocking. The first year alone requires 6 treatments with a staggering price of $125000 per treatment.  That’s $750000 for the first year alone. Every subsequent year requires 3 doses of the drug administered through a lumbar puncture. This excludes administration, hospital and professional services costs.