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Treatment for families battling a devastating rare disease

Treatment for families battling a devastating rare disease

New Zealand infants suffering from a rare neuromuscular condition may soon be eligible for compassionate access to a life-changing new medicine.

Spinal Muscular Atrophy (SMA) is a rare genetic disease that causes muscle atrophy, weakness and shortened life expectancy. There are four types of SMA which vary in age of onset and severity, but 95% of children with SMA Type 1 will die within 18 months.

In recognition of the urgent need for treatment, pharmaceutical company Biogen will provide SPINRAZA, approved by the FDA since December 2016, free to New Zealand infants with SMA Type 1, the most severe form of the condition. The drug will be administered through regular spinal injections at Starship Children’s Hospital and has been shown to significantly improve motor function enabling affected infants to achieve milestones.

“Being given compassionate access to this drug is a huge win for families coping with this devastating rare condition while we wait on registration and funding,” says Ronelle Baker, Chief Executive of the Muscular Dystrophy Association of New Zealand.

The drug SPINRAZA is being marketed around the globe by Biogen and has recently been funded in Australia for the treatment of children with SMA Types 1, 2 and 3a who show symptoms before three years of age. Biogen have applied to MedSafe to register SPINRAZA in New Zealand and will follow up with an application for funding to PHARMAC.

“It’s really tough to get medicines funded for patients with rare disorders, and New Zealand is slipping further and further behind other countries for access to medicines,” says Dr Collette Bromhead, Chief Executive of the New Zealand Organisation for Rare Disorders (NZORD).

NZORD and MDANZ have been working with Biogen Australia to get this life-changing drug available for all patients with SMA in New Zealand by supporting the MedSafe application and advocating for funding through PHARMAC.

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