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  • he spent his whole thing.

  • Man has spent 17 years battling to save his Children from an incredibly rare genetic disease and find a reliable and approved treatment.

  • And eventually he did, in the form of an unusual product.

  • We killer Knicks Zeros Sons have what's called black bone disease, formerly known as Al Captain Yuria or a k U for short.

  • So aka you is an ultra rare genetic disease effects Roughly one person in half a million on it's due to a mutation in a gene, which means that patients can't break down a certain acid that accumulates in cartilage and bone and goes black, which is why it's called black bone disease.

  • And over the years that leads to severe joint deterioration, spinal problems, heart problems and intense pain.

  • Ciro's eldest son, Julian, was diagnosed as a baby, but symptoms can be delayed until the patient is an adult.

  • He's now 20 as a patient at the National Al Captain Nouria Center in Liverpool.

  • He has had early access to a weed killer derived drug called night is unknown.

  • Scientists knew United Simone should help prevent the damage but needed to prove it.

  • Using large scale clinical trials mhm, but with only 1000 known suffers worldwide, there was no help from big drug companies.

  • So 10 years ago, zero gave up his job to concentrate on the campaign.

  • He's raised millions of pounds.

  • The research did prove notice in own works, not only stopping symptoms developing but even reversing some of the damage done.

  • It has now been licensed by the European Medicines Agency on for the last three years while taking notice unknown, Julian has been symptom free.

  • If I wasn't on the drug, it could be that within the next 10 years it will likely within the next 10 years.

  • I would definitely feel the effects, Um, in my joints.

  • Uh, possibly my heart valves, um darkening or like dark spots in the eyes is just everywhere there's cartilage or anywhere that something called a Kronos ISS can form the A.

he spent his whole thing.

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B1 drug disease cartilage bone julian genetic

A father's fight for a drug to treat his kids

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    林宜悉 posted on 2021/01/16
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