Muscular Dystrophy Ireland is today urging the Minister for Health Simon Harris, and the HSE to approve funding for a life changing drug - Spinraza - for children with Spinal Muscular Atrophy (SMA). The call comes as families from the SpinrazaNOW campaign who made an appeal at Leinster House today.
SMA is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement, causing muscle shrinkage and wastage. SMA involves the loss of nerve cells called motor neurons in the spinal cord and is classified as a motor neuron disease.
Although classified as rare, SMA is the leading genetic cause of death of infants and toddlers. Without treatment approximately 95% of babies with Type 1 diagnoses do not survive past the age of 18 months. Children with Type 2 SMA face the prospect of progressive muscle wasting, leading to an inability to walk or stand unaided, scoliosis, problems with breathing, feeding and failure to thrive.
Elaine McDonnell, Chief Executive Officer of Muscular Dystrophy Ireland said:"Spinraza has proven to be one of the more successful therapies to emerge in recent years, reducing the muscle wasting effects of Spinal Muscular Atrophy."
She said:"It has already been approved in many other European countries, and has already been made available to children with SMA Type 1, through an expanded access programme. We now need to see the treatment extended to all children affected by SMA, given its life saving impact for children with SMA Type 1, and potentially life changing impact for those with SMA Types 2, and 3. We believe that this decision can have a hugely positive impact on the lives of our members and families who would be eligible to receive Spinraza, and further benefits other emerging therapies in the future."
Ms McDonnell said:“We have travelled a long road to get to where we are, from the families to the medical experts involved and their teams, as well as MDI staff and representatives. They have all worked extremely hard up to this point and those efforts deserve to be rewarded with continued and expanded access to new and innovative treatments.”Despite an initial decision not to recommend Spinraza the Commercial Pricing Unit (CPU) of the HSE in late 2017, there is hope that Minister Simon Harris will move to approve the reimbursement of Spinraza for patients with SMA following his commitment to the BeNeLuxA Initiative last June. The initiative seeks to improve long term access to drugs like Spinraza at affordable costs, and mandates national experts to participate in the activities of the Initiative. Members Belgium and the Netherlands have recently approved the reimbursement of Spinraza, and it remains to be seen if the Minister’s and HSE’s decision will be consistent with Ireland’s commitment to BeNeLuxA.
Already available across much of Europe, patients in Italy, France, Germany, Norway, Finland, Austria, Switzerland, Spain, Luxembourg, and Slovenia, as well as Israel, Australia, and Japan while in Denmark, infantile onset SMA patients have access. The HSE’s failure so far to approve Spinraza is one that is inconsistent with the actions of other European and International health systems, and will have a potentially devastating impact on patients and their families.
Sinead Perez is the mother of 10 year-old Jordan Perez, from Sixmilebridge, Co. Clare.
She said:“Spinraza is more than a drug. It brings hope to people who have never had hope before. Until now it's been a slow march downwards, watching someone fight with their own body as it fails them more and more."
She said:"Without Spinraza Jordan's condition will continue to deteriorate. He's losing muscle function every day that passes. Spinraza will help maintain him at his current level or even help Jordan to regain some of the muscle function he's lost. It may give him back some of his independence taken from him by this deadly disease, like the ability to feed himself, scratch his head or lift a cup, simple everyday tasks we take for granted. It will give Jordan the chance at a longer and higher quality of life by helping to slow down, stop or even reverse the progression."
She said:"This is the hope it brings, and more, it also brings the hope that Jordan will have the chance to grow up. To make it to adulthood, where he can use his talents to contribute to society like everyone else. What mother would want anything less?"
For more information on the #SpinrazaNOW campaign, visit www.smaireland.com
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