The story

The 1992 Hollywood movie "Lorenzo's Oil," depicts the true story of Lorenzo, a five-year-old boy who suffered from adrenoleukodystrophy (ALD), a rare and incurable disease that slowly destroys the entire nervous system.

The movie showed how Lorenzo's grave physical and mental decline was finally stopped when his tireless parents found a treatment based on a mixture of oils, despite skepticism from doctors.

The film illustrated perfectly the struggle faced by patients suffering from any of 6,000 known rare conditions worldwide, generally known as orphan diseases.

Michaela Damin, founder of the Barth Syndrome Trust, whose son, Nick, suffers from this crippling syndrome that affects the heart and immune system and only has 130 known sufferers worldwide, told CNN: "It can take years to get the correct diagnosis and even then there may be no expert who knows what to do next."

The fact remains that rare diseases are still mostly overlooked by pharmaceutical companies because of the small patient population and lack of financial incentives. Read full article »

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