LET LIAM LIVE - AN UPDATE

We have had two replies from Liam's Dad both of which show what a humble and intelligent person he is. In the first one he is keen to say that they do not wish to attack ISIS pharmaceuticals but would rather try persuasion. We have made some suggestions.

In the second he replied as follows

Hi there, 

Thank you for your support, your encouragement given us a great ideas, I think, we now have a plan to make something big.

Obviously we will work quickly, because each day count in the SMA. In waiting if I understand we need to put into our communications the #smashsma ,
#letliamlive and the link to your blog?
Give us a few days and the Canadians will be to the appointment:-)

 

I replied with

 

 

Excellent. You can use any of our information. You can use #smashsma #letliamlive or our blog if it helps. I will write to or speak to anybody you suggest. I will do every single thing I possibly can to help you. It is essential that your son is given a chance. You have my 100% commitment to your cause. I have contacted a friend in the American Embassy. I think you are right it is proper to be polite with ISIS but they will listen better when you and that lovely boy are on television. Take care and - I hope this sounds proper - despite all of these things enjoy every moment with your little fella because these are special days that will not come again. You know that I am sure but I wanted to say it.

 

Tom  

CAN WE SAVE THIS LIFE ?

THERE IS AN 8 MONTH OLD BOY AND HE NEEDS YOU- PLEASE READ


You may have seen an article about a little boy called Liam who is 8 months old and has SMA. The trials that we have been fighting for are STARTING in Canada and SMA type 1 babies are being given a chance to live - apart from Liam because he is 17 days too old to qualify for the trial. You may have heard this story - well today we received a direct appeal from Liam's parents asking if there is anything we can do.

Please can I ask you to make a fuss about this - let's make a noise all over TWITTER. The hash tag will be #letliamlive LET LIAM LIVE. Even if he is outside the age for the trial he should be allowed the drug and the results should then not be included in the study. There are not thousand's of these babies there is this one little lad and they are going to let him have no chance of regeneration because he is 17 days too old.

This is barbaric.

Here's the story




LET LIAM LIVE


MONTREAL - An infant who's slowly dying of a rare spinal defect has been denied an experimental medical treatment because he's 17 days too old.
The cutoff set by the U.S. pharmaceutical firm ISIS has outraged his parents, Yan Defosses and Emanuelle Desbiens.
"It's cruel and completely inhuman," Defosses told QMI Agency.
Eight-month-old Liam suffers from spinal muscular atrophy, a genetic disease that attacks the spinal cord, weakening his muscles.
His parents realized something was wrong soon after his birth. He was losing muscle tone instead of gaining it.
When he was four months old, doctors gave his parents the diagnosis and said the baby only had two years to live.
He's already showing symptoms of the disorder. He can't lift his head without help and has difficulty breathing.
But the family's hopes were kindled when they learned that California-based ISIS has conducted clinical trials on the drug SMNRx.
"We are in contact with families in the United States, and it has completely changed the lives of their children," said Defosses. "They've survived under more than acceptable conditions."
Earlier this spring, the company announced a second phase of clinical trials, this time in babies. But the children had to be seven months old or less before May 1. Liam was seven months and 17 days old.
Liam's doctors said they could do nothing because the trials aren't being conducted in Canada.
Saint-Justine Hospital and Health Canada were prepared to approve Liam's treatment but ISIS refused.
"It shocked us and destroyed us," said Defosses.



Below is the heart-breaking plea from his Dad

Please tweet about this - let's start the ball rolling

Please tweet ISIS - Please allow Liam to be included in your SMA study. Please let hum live #letliamlive



-------- Original Message --------
Subject: Message via votre profil Google : We need your help
From: Yan Défossés <yan@groupeinfiny.com>
To: estellameansstar@gmail.com
CC:

Greeting to you,, my name is Yan I'm the father of a child who are diagnosed with sma type 1. I write to you from Montréal Canada, like you probably know Isis pharmaceutical begin a clinical trial on infant with her compound the SMNRX. Our son Liam meet all the criterias for this study, unfortunately the canadian centre is not yet rolling and my son just turn 8 months. So he is now too hold for the study.

Because we know how it's important to restore as soon as possible the smn protein level, that few weeks, can make all the difference to restore motor function of our child, we continued our research, to avoid delaying access to the experimental treatment. After describing the context and give all the information we have about Isis and SMNRX, to Health Canada, equivalent of the FDA in U.S. we obtain a special authorization granted by Health Canada allowing Isis to provide the drug out off protocol and releasing any liability. With that way, Isis can receive more data and is not obliged to diffuse or account the result in the actual study.

Obviously, there is no obligation for Isis, to accept. And there is the point, she refuse:-( I understand you make some pressure last year to stress Pfizer to invest in the SMA and it seems that it work with her engagement in the RG3039 and Repligen. Could you give us advice to encourage them to change their minds quickly, before the therapeutic window of our little boy has passed? Thank in advance The Desbiens-Défossés family tel: 1.514.583.1247 Thank you so much again for all you do for the SMA and sorry for the strange writing we usually speak french at home Yan



I have replied

Yan

 

I was devastated to read your story. Our little girl died aged 8 months from the same disease and we have been working since then to get drug trials and now we are told that ISIS say no to your case. The argument you put forward is fair and reasonable and should appeal to rational and caring hearts,

 

BUT

 

What you need now is to make a noise

 

1) We have asked 44,000 twitter followers to tweet their protest at ISIS and we are using the message LET LIAM LIVE #letliamlive

2) We will do the same of FACEBOOK

3) Messages are being left for you on our blog at www.smashsma.blogspot.com

 

BUT

 

You are in Canada. Get the local TV station. Get the local newspaper. Get the local radio. Go and sit in the lounge at the TV station until they will hear you and put you on TV. this is a heart-breaking story and they will give you publicity - publicity enough to SHAME ISIS into acting. You need to tell then that thousands of people in the UK are backing you and you need the people of Canada. Liam needs the Repligen  and he needs it now. If the facts are not included in the overall trial so what it is a moral and kind act - it is a human act to give the boy the help he needs.

 

If ISIS will not listen then get your friends and your family to sit in their reception until the TV cameras turn up. You are fighting for the quality of life of your treasure and you must do everything you can. It is not enough for them to use lame arguments about access to the drug. there are not hundreds of babies with the condition so the argument that they have to draw the line somewhere is NOT ACCEPTABLE. I will do anything at all to help from this end. Do you have a TWITTER account ? Please let me know. We are with you - we are praying that sanity sees its way through. Please let us know what happens. please send more photos of Liam to help me spread the word at this end. I lost my little girl and we swore to her that we would SMASHSMA - well she would love it if this became a very concrete example of her legacy. the people here are amazing and they will be writing to you and giving their hearts.

 

Be strong. Fight



Emmanuelle Desbiens · Polyvalente de Lévis

We are the Liam's parents and we would like to clarify, that although it may seem cruel, we understand and respect the position of the pharmaceutical to strictly follow the eligibility criteria of their study. Our sadness is that Isis refuses to grant the three experimental doses despite a special authorization granted by Health Canada, the equivalent of compassion access the FDA in the United States. The authorization provided by the Special Access Programme to drug released from all responsibilities this pharmaceutical. Isis could collect additional data without creating noise in the current study. We follow the developpement of this compound and be confident its the good for our child. In addition, if you know this disease (SMA TYPE1) you know that now is the time or never to help.