Hope for Stephanie

FROM just $6.40 a script, a Moriac family will now have access to a previously unaffordable life-changing drug that is the only known treatment for their daughter’s muscle-wasting disease.

Stephanie Nave (pictured) was diagnosed with the incurable Spinal Muscular Atrophy type 2 when she was 17 months old.

The rare, inherited disease destroys motor-neurons and means even a mild cold can seriously impact Stephanie and her ability to move.

Late last year the Therapeutic Goods Administration approved the only known treatment for the disease. But, Spinraza (nusinersen) was not covered by the Pharmaceutical Benefits Scheme. It had a listed price of $1million for a 12-month dose, and more than $360,000 a year for subsequent doses. But, from June 1, Spinraza will be available on the benefits scheme for just $39.50 a script or $6.40 for concession patients.

Stephanie’s parents, Amanda and Mark Nave, have been publicly campaigning for the drug to be listed on the scheme for close to a year.

The Naves never lost hope, even when a listing application was knocked back in December. After that devastating announcement, Dr and Mr Nave travelled to Canberra to meet with the Pharmaceutical Benefits Advisory Committee and share Stephanie’s story ahead of a resubmission.