GOOD LUCK HAMISH!
You may have seen Hamish around the school yard getting around in his wheelchair and wondered why? Hamish is often stopped and asked what has happened to him, and when out he is often the subject of uncomfortable stares by people, who seeing a child in a wheelchair find this confronting.
Hamish has a rare “inherited genetic muscle wasting disease” called Spinal Muscular Atrophy. “This is characterised by a loss of nerve cells called motor neurons. The affected muscles not only include the muscles you see like your arms and legs, but all your internal muscles like breathing, coughing and swallowing” (SMA Australia Website). SMA is the leading genetic cause of death in infants and toddlers around the world. SMA is an inherited genetic condition.
In the last twelve months, a drug that has been developed by Ionis Pharmaceuticals and licensed through Biogen Pharmaceuticals, has been approved through the FDA in America and more recently the TGA and PBS in Australia that will stop the progression of the disorder. The drug called Spinraza has had significant results with a range of SMA patients, including new born SMA Type 1 patients who are reaching milestones, instead of degenerating and dying due to the muscle wasting effects of the disease. This drug has provided incredible hope for children and adult’s with SMA as well as their families.
Hamish who has type 2/3 SMA is beginning the journey with access to Spinraza TOMORROW! Our hope is that he will begin to gain strength as he grows rather than become weaker.
The Federal Government has been relatively quick in the response to the approval of Spinraza for Children with SMA and it took only two reviews through the Pharmaceutical Benefits Scheme reviews panel to approve it. The task for the community now is to have it approved for adults who are living with SMA.
We wish Hamish good luck tomorrow and will keep him and his family in our thoughts as we hope you will too.