1 March 2014 - There's one thing worse than suffering from an ultra-rare deadly disease with no treatment — having an ultra-rare deadly disease that has a treatment you can’t afford.

Hundreds of Australia’s sickest people wake up to this reality every day.

The Federal Government’s funding for orphan drugs has slipped far behind that of other Western countries.A rare strain of cystic fibrosis has a treatment, but it’s stuck in bureaucratic limbo. Deadly auto-immune disease aHUS has a treatment that saves the lives of those given access, but it’s yet to be approved for funding on the Life Saving Drugs Program.

There is a drug that halts the progression of the debilitating and life-shortening Pompe disease, but only children with the disease who are diagnosed at under 24 months of age can access the treatment. This excludes a 12-year-old Craigieburn boy because he’s considered to have “adult-onset” Pompe.

For more details, go to: http://www.heraldsun.com.au/news/victoria/anger-as-lifesaving-drugs-stuck-in-federal-bureaucratic-li...