Saving Ashley: What price a life? | Adelaide Now -g-icon-error cloudy-day nav_small_right nav_small_right nav_small_right nav_small_right nav_small_right nav_small_right nav_small_right nav_small_right nav_small_right 0A0871E9-1636-49F4-9041-2E36E2BB5333

He is only five years old and cannot pronounce a deadly disease.
The only drug that can save him will cost $240,000 a year, but who should pay?
It\'s a cheeky battle, gap-toothed, puppy-
Special little boy who loves to live.
Gray almost dumped the hinge of the front door. “You’re here!
Look, I lost my first tooth.
\"He jumped from his right foot to his left.
He smiled so brightly as if he had laughed twice.
Not long ago, he took a picture with a children\'s model agency --
They got three times in the third photo taken for the other kids.
It\'s Ashley\'s.
His big blue eyes are always the last.
Ashley is different from the other five. year-olds.
He suffered from a rare and incurable fatal disease.
In the first year of his life, it almost killed him.
He\'s here now-
In the corridor of the little house his parents rented, jumping over and over into the air --
Just because of the charity of a pharmaceutical company.
He\'s taking drugs that the federal government won\'t fund.
The 300 mg he gets every two weeks is the cost of a family car.
$240,000 a year.
He is healthy with it.
Without it, he will die ill.
That\'s simple.
But the terrible reality is that Ashley\'s future depends on the goodwill of multinationals.
Or the bureaucratic pen and the nod of the politician.
So every time he takes liquid gold, his parents silently pray for thanks.
There are still two weeks left for a priceless son.
The lower bunk is Ashley\'s.
He shared a small bedroom with his two brothers.
Baby Ollie in the little bed in the corner
Lachlan has a bunk.
A fluffy Elmo blanket lies on Ashley\'s bed.
The blanket came out when he found the liar.
It could be 40C outside-
One day in eastern Melbourne, he will drag it to the lounge.
Curled up on the sofa.
It was the first signal that Jason and Kerry Gray\'s son were hospitalized again.
The medicine he took was flawed.
He is prone to severe infection and if he has a fever, he will go directly to the hospital to check and use a lot of antibiotics.
Ecullizumab prevents part of the immune system from working against brain and blood poisoning.
They can\'t take risks in the heat.
With it, however, Ashley can grow old now.
A smart mind can\'t find a way to treat aHUS, but this unique method of treatment controls his immune system well.
Australian authorities do not fund ecullizumab for aHUS.
A year ago, it was rejected for inclusion in Australia\'s life-saving drug program.
It does not pass strict standards.
This could happen again when a panel of experts again next month considers whether the Minister of Health should be advised to approve.
If so, it could change the lives of up to 70 Australians.
There is no guarantee that Alexion will continue to fund Ashley\'s treatment.
The global giants have recently rejected many requests from Australian patients who are sympathetic to the need for them.
They are waiting for the federal government to start paying.
When they wait, people get weaker and weaker.
15 cm deep outside the constate office window in Steve spinto.
The condition below freezing and Blizzard is 6C.
A little boy, who helped change his life, is now running around 18,000 km m away in shorts and T-shirtsshirt.
Ashley is one of hundreds of people in the world.
The drug works very well and governments in 40 countries are funding aHUS patients.
Some call it Steve Jobs in the biotech world.
But for years, the pharmaceutical company he worked
It could be a failure of $1 billion.
This is how much ecullizumab has invested.
In the 1990 s, researchers at Yale University came up with a potential way to stop the immune system from rampant, and they turned their attention to treating big diseases.
It looks like it\'s gone.
After two and a half years of building a small lab, there is still nothing to show.
Ten years later, there was only a limited victory. It looked grim.
But a British blood expert insisted that he wanted to use a super
Rare autoimmune diseases have finally paid off.
Nearly a decade after Alexion began, the breakthrough came.
Squinto remember very clearly.
He finished his last supper with his wife. The phone rang.
It\'s a call from Britain.
The result came out.
11 patients in the trial are improving.
There was a major illness on the brakes and the Alexion team turned their attention to a rare disease.
They started applying it to patients with Ashley syndrome.
It works very well too.
Squinto thought of selling the company for its brighter prospects, but he and his colleagues
Founder Leonard Bell decided to solve the problem completely.
\"We\'re not real hands-it-
\"That\'s the kind of guy,\" he said . \"
Over the next few years, after being approved for aHUS treatment, doctors from all over the world called.
Some families are begging companies to allow them to participate in their charity programs.
No one can afford it.
Joshua Cosman, a kidney expert at the Royal Children\'s Hospital, is one of them.
He first met Ashley in 2009, who was very ill.
Ashley is a candidate for the drug ecullizumab (or Soliris because it has been sold) because he continues to start and, despite their efforts, he is further stuck in kidney failure.
Kausman recalled: \"He is at a more serious stage of the disease.
But we still have a chance to reverse it with ecullizumab.
\"Within a few weeks after starting taking drugs, Ashley\'s condition has improved.
Before the new year, a black Labrador puppy, Disel, galloped at Ashley\'s foot on the back deck.
The children gave him a gift for Christmas.
The boy jumped around him, a wish for Santa Claus.
This is at the top of his Christmas list.
When Ashley tried to recite the name of his illness --
Syndrome of atypical lysophanide
The diesel bit his ankle.
He hit fal and said, \"there is anemia in urine.
\"It was a bold effort, a medical attempt for a little guy.
He did perfect a few years ago.
He forgot that it was a good sign.
It\'s been too long. his illness has cost this family.
Mom, dad and five children, including the Tahlia Sisters and The Mikayla sisters, who live with the children in the room in the lobby.
This unpronountable disease is no longer talked about.
Now Kerri and Jason () can value simple things. And they do.
Spent a family day at the beach with dogs.
Camping in RV Park.
Backyard full of green grass.
The noise of small feet and sound.
The swing squeaks back and forth with one child or another.
Kerri smiles and celebrates his son\'s birthday with tears.
Five have passed, but those celebrations have not lost their luster.
The beginning of his life was so hard that she didn\'t even believe he would see him for the first time.
For Australians like Ashley, language has changed everything. “Substantially.
\"After a review, it was included in standard 4 of the life-saving drug program in 2009.
This is a major change.
People with rare diseases think it helps push the project --
Life-saving drugs
They can\'t reach it.
All promises but no delivery.
Standard 4 now stipulates that a drug can be accepted by the program, and the Drug Benefit Advisory Committee must \"predict that the life of the patient will be greatly extended due to the direct consequences of the use of the drug \".
In the field of medicine, rigor is everything.
\"Extending life significantly\" really means Megan Fox, who has a rare illness and thinks the word makes the process less objective. More ambiguous.
She is in charge of a growing force for rare disease propaganda --
A rare sound in Australia.
Ashley is just one person represented by her team.
But so far he is luckier than most people.
There is no doubt that his free medicine provides him with a longer life span.
Much longer.
He was the simplest baby at first.
He always sleeps and eats like he should.
The happiness of a busy mother.
Kerri\'s mother\'s focus was built on the wall of their home
When the children were less than five months a week, she made molds with the hands and feet of each of them.
Despite the increasing number of chicks, everything is over.
Two months after Ashley\'s feet and hands were caught in plaster, he became uneasy.
Something triggered Kerri\'s sixth sense on a Sunday night, and she took him to a new medical center to worry about a serious problem.
\"Jason asked me to give him a breath and put him to bed.
Her husband sat next to her on the sofa and smiled shyly.
The general practitioner suspected that he had anemia.
They asked her to take him to the emergency department at Box Hill Hospital.
The consultation room was packed with medical staff.
Chaos, hurried conversation, inserted drops, nurses running around with stainless steel trays full of needles and tubes.
And the eyes of mercy.
\"This is the first time I \'ve been to \'Oh, s. .
This is serious, \"said Kerri.
\"They sent him to the Royal Children\'s Hospital in an hour --
At the same time, I\'m still worried about small things like how I should handle the stroller when I\'m in an ambulance.
She wants to know how unimportant these things really are.
AHUS became everything in the next year.
As soon as the diagnosis was confirmed, Ashley received routine treatment for aHUS patients.
But it only works in part.
He had dialysis and plasma exchange.
But his kidney function is down.
It doesn\'t look like Ashley will grow up.
The most important thing for her is how much they will pay if she and Jason have to pay for the magic drug to keep Ashley alive.
This is impossible.
This is the most expensive drug on Earth.
Although the price is expensive, it is worth considering that every taxpayer in Australia will lose only 1 pound.
To keep Ashley alive, nine cents a year.
If each known aHUS patient receives a $2 Grant.
Each taxpayer pays the most every year.
Because it does not work for every patient, it is likely to be much less. It is not much.
But for these people, their lives are worth it.
It is called orphan medicine.
They are usually expensive because they are developed for ultra-niche markets
Patients with rare diseases
That\'s why the public health system is easier to digest.
So very few people actually need to use them.
Meghan Fox said that although the Australian health system has conducted more drug research on rare diseases, it does not support their success once the results come out.
This is a sign for her that this small, rare disease cohort is considered the highest level of second-class population.
Her hopes were dashed last year.
The review of the LSDP was announced a month before the federal election.
About 20 people attended a conference call.
Department officials and delegates of rare patients, drug companies and doctors.
There is a cautious but hopeful buzz on this line.
But nothing since then.
No email, no phone.
Fookes is still hopeful.
\"I welcome comments. We need it.
\"She should be a member of the review team.
\"We are worried that the LSDP will get worse.
\"Australia\'s well-known rigor in medical research seems to have put funding for rare diseases in trouble.
Orphan drugs often fail in tests on projects because the rare disease population is so small that they often fail to meet the harsh dual needs
Blind clinical trials of common disease drugs.
The doctor ventured that if the method did not throw the baby out with the bath water, it would get closer and closer.
And Fookes are confused.
\"For some reason, patients with rare diseases must defend their lives.
People like Ashley don\'t get sick from overeating, smoking, or taking illegal drugs.
They were born with this disease.
But in terms of the federal government, this is simply bad luck \".
The day they learned that Ashley might die, he arrived at the hospital from Kelon\'s job.
Why didn\'t he get the speeding ticket? it was a good luck.
He was sitting next to Kerri at the Royal Children\'s Hospital and Kausman explained the test results.
In that room, Kausman told them something like this: Part of Ashley\'s immune system --
\"Supplement \"-
Causing uncontrolled action.
How could they not know?
Probably a genetic defect.
It causes the blood vessels to contract, causes the platelets to form a clot, and puts physical pressure on his oxygen
Carry red blood cells that are splitting.
He needs dialysis.
There is no cure.
The disease is fatal.
Their son could die.
But all Jason Heard was chaos.
None of this makes sense.
These words will not be said.
\"I remember sitting there with the doctor and turning to Kerri and asking her,\" Kez, what happened, \"she just cried and said,\" I don\'t know, jase.
I knew it was serious.
On that day, the critically ill boy was evaluated by the Hematology, kidney disease, immunologist, surgeon and pediatrician.
Before six o\'clock P. M. , he was doing surgery to prepare for dialysis.
Compensation for kidney failure
Jason works for a mattress company so he can take care of their three children at home.
Ollie was born three years later.
Kerri and Ashley spent a year in the hospital.
She worked on the autopilot for a couple of weeks at a time, then it would pour in the pile for a day, pick it up again and reset it to the autopilot.
Jason is like a single parent to other children.
For a while, he was at a distance from the sick boy in the pool --blue eyes.
\"Deep in my heart, I think I must have decided if I didn\'t get close. . .
Jason shook, \"it will be easier to lose him.
\"The data doesn\'t look good for the government. Since 2009 —
When inserting \"substantial --
Only one new disease group has been added to the list of beneficiaries of the program.
In any case, the group has reached 80 through the approval process.
Another drug has also been added, but this is an update on the disease that has been reported before 2009.
Dr. Suzanne Hill, chairman of the pharmaceutical Welfare Advisory Committee, declined to be interviewed on the life-saving drug plan.
She replied politely that it would not be appropriate to answer even general questions with ecullizumab --for-
Waiting for aHUS\'s decision
A rare disease representative, who declined to be named, told the Herald Sun that the meeting with Hill was particularly frustrating.
\"She seemed to sympathize with us when she heard what we said.
But in the end, she said, \"you need to talk to those politicians, not to me . \"
Kerri Grey, who set up the aHUS support group in Australia, believes that the government can spend more money, but not.
Dollar data has been overshadowed by broader projects.
\"The plan is demand --
Drive as needed, \"Professor Chris Baggoley, chief medical officer, said in an email in response to an interview request.
The idea of the program is to allow access to targeted rare disease treatments that cannot pass the cost of the Pharmaceutical Benefits programbenefit test.
Fookes says valuable aim has been diluted over time.
\"As there are more and more drugs to treat rare diseases, it is getting harder to list them,\" she said . \".
\"I think they want to slow down the project, but they basically blocked it on its track.
His parents saw how close he was to the cliff.
Before Christmas, Kerri had a crazy contact with a Queensland mother.
She often hears Ashley\'s story without ecullizumab.
Bianca Scott is lying on a hospital bed in Gold Coast.
She does dialysis for hours a day to deal with her failing kidneys.
She has plasma replacements trying to shut down her crazy immune system.
Treatment does not work.
Three weeks ago, her mother Tami Hamawi listened to her daughter.
Her lungs were full of air while she was sleeping.
The last thing she thought of that night was probably the loss of her only child.
Bianca had aHUS when she was a baby, but it suddenly eased.
Looking back at some of her graduation photos in October, she looked pale and beautifully painted.
This is the beginning of her rapid decline.
The Bianca family has this disease.
Some relatives who died of kidney disease decades ago are now known to be caused by aHUS.
Ecullizumab is beyond the reach of children.
She asked Alexion for a discount but she refused.
The kidney expert told her that Bianca had two months before her kidney damage was irreversible.
She is now trying to find thousands of dollars for her girl\'s life.
Experts think, politicians delay, but children like her slides.
Tammy is still an optimist.
She hopes that Bianca may be the first person in her family to break the curse of aHUS.
But she\'s running out of time.
There is hope.
Darts at the front and corner of the Royal Children\'s hospital corridor.
When his mother found him again, he was leaning very close, pulling down his lower lip and showing the gap in his teeth to the cleaner who squatted down to chat.
He got $5 for it.
There is also a loose one next to it.
Just $10 a week.
The RCH staff has seen Ashley fall into a trough with his illness, the founder of invasive therapy, and then bounce back with his $9000 --a-dose drug.
The visit to the tooth fairy hearing their little patient is heating up.
Children should have enough mood to celebrate these small things.
One nurse said that when Ashley gets an infusion every two weeks, they like to be rostered.
No fuss.
He smiled T-
The shirt, let the drip connect to his portacath, right under the skin on the left side of his heart.
He doesn\'t know much about his illness.
His \"special medicine\" is a part of life, just like eating vegetables for dinner and brushing your teeth.
He doesn\'t know how close his parents are to losing him.
He doesn\'t know how much his life is worth. Nor should he.
Instead, Ashley Gray fell asleep in his car seat on her way home from the hospital.
His head tilted to one side.
When he grew up, he dreamed of becoming a dolphin, an Iping puppy and Slinky springs.
And how he will spend $10 in wealth.
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