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Saturday 20 October 2018

HSE urged to fund spinal nerve 'wonder drug' for babies

Temple St Children’s Hospital
Temple St Children’s Hospital

Muscular Dystrophy Ireland (MDI) is urging the HSE to approve funding for a "wonder drug" that could save the lives of 95pc babies affected by an ultra-rare condition.

The advocacy group's call comes following the successful treatment of two Irish babies last year with Spinal Muscular Atrophy (SMA).

SMA is a genetic disease affecting the part of the nervous system that controls voluntary muscle movement, causing muscle shrinkage and wastage.

SMA involves loss of nerve cells called motor neurons in the spinal cord and is classified as a motor neuron disease.

According to MDI, the disease is the leading genetic cause of death of infants and toddlers.

"Without treatment, approximately 95pc of babies with Type One diagnoses do not survive past the age of 18 months," a spokesperson said.

"Children with Type Two 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."

Infants

Last January, two infants from Dublin were said to be the only two babies in the country with SMA. However, after hearing about their case, pharmaceutical company Biogen gave them a second chance at life by offering Temple Street Children's Hospital a chance to take part in its expanded access programme for its "wonder drug" Spinraza.

"Spinraza has proven to be one of the more successful therapies in recent years, reducing the muscle wasting effects of Spinal Muscular Atrophy," said Muscular Dystrophy Ireland chief executive Elaine McDonnell.

"It has been approved in many other European countries, and has been made available to children with SMA Type One, through an expanded access programme.

"We need to see the treatment extended to all children affected by SMA, given its life-saving impact for children with SMA Type One, and potentially life-changing impact for those with SMA Types Two, and Three.

"This decision can have a hugely positive impact," she added.

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