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Why Do They Fight Like Lionesses?

I have read a lot of articles in the previous two or three weeks. I have learnt a lot of wise things and a lot of foolish information as well. Within that time there has also been lots of hope, disappointment and broken promises from the Government Office. After going through a huge amount of sources I have learned the truth and I would like to share it with you. Why? The reason is very simple. Mothers of children suffering from SMA type 1 are often presented as women who want to experiment with the lives of their own children. And do you know what the truth is? They are actually trying to save them because it is the most important thing the mother can give her child – give life and sometimes give it even two times. And this is what we are fighting for. It is not about the fact that there are “only” four children and a very expensive treatment. If we cannot unite and fight now how do we want to make this system change? The ill and defenseless are the most vulnerable ones and if we “the healthy” do not start fighting for them – who will fight for us?

On February 12, 2020 Prime Minister Peter Pellegrini initiated a meeting with parents of children suffering from SMA that he did not take part in. This meeting with mandated personnel of Ministry of Health was a huge disappointment. No, it is not all about the money. Indeed we and the mothers as well understand that the price AveXis Company charges for Zolgensma treatment is incredibly high. We talk about the way the government treats this issue. Do you know how? Very evasively. Actually they did not give a helping hand at all. They allowed delivery of the most expensive treatment that can save these children´s lives but only under the condition that the treatment will be managed by a doctor who will take all legal responsibility for any side effects. It means that the permission given to the parents was immediately taken back.

But this is not what I want to write about. My goal is to explain the reasons why these mothers fight like lionesses so that their children can get the one dose of Zolgensma treatment.

There is a treatment called Spinraza approved in EU. It is a great treatment and when it was launched onto the market it was a number one treatment. But it was not perfect. Spinraza is a treatment that contains nusinersen. The treatment helps to create proteins necessary for development of muscles. But this treatment does not cure the disease, it only slows it down. Another thing is that Spinraza is an intrathecal injection applied in lower spine (lumbar puncture) near the spinal cord. The treatment must be applied in a series of six effective units in precisely specified period. Afterwards it is applied once in every four months for lifetime).” See https://www.vozickar.info/spinraza-je-pripravena-pomahat/) One dose costs 81 876.61 € in Slovakia (see https://www.adc.sk/databazy/produkty/detail/spinraza-12-mg-injekcny-roztok-629836.html ). Adding it up the total annual cost is 327 506.44 €. Actually it means that Spinraza is a treatment that slows down the course of the illness and it is much more expensive than one dose of Zolgensma. Why? I am glad I can explain that. Spinraza (as it is stated above) must be applied for lifetime. So if we multiply the annual costs with number of years the treatment will be applied (in this case it relates to four children), the amount of money paid for Spinraza is much higher than for one dose of Zolgensma.

„And for that reason introduction of Zolgensma compensated these prices when curing SMA,“

said Dave Lennon, president of AveXis for POLITICO.

And we get to the main point. And it is the price of one dose of Zolgensma. The price is remarkable- 1.9 mil €. I know, this number is astronomical but in comparison with Spinraza (that is actually more expensive in long term period) and for the ill children it is redemption.

Do you ask  why?

In every human body there are various genes including gene SNM1. It is a very important gene responsible for the fact that we are walking, moving hands, that we can eat and yes- that we can breathe.  The gene SMN1 creates a protein called SMN that controls motor neurons. So in different words – if we have this gene everything is all right. If we miss it our muscles are getting weaker.

I could compare it to a broken leg in plaster. When the plaster is removed after a month or two, our muscles are weak, tough and unable to move. For that reason we have to undergo rehabilitation that can last months … including the pain.  But the situation with the missing SMN1 is much more dramatic. Children suffering from this disease cannot recover their muscles from rehabilitation. Muscle cell death is a permanent state and consequences are disastrous as after some time they are not able to eat or breathe without assistance. And actually Zolgensma is a gene therapy applied as a one-time infusion to the whole body so the application differs a lot from the painful lumbar puncture. This way the treatment gets to the whole body and replaces the missing gene that helps to produce the very important SNM protein.

When Zolgensma was launched the price of the treatment faced lots of criticism from EU governments. And we understand that that strong emotions and desperate efforts of mothers to save their children are the best way to make business from this kind of situation. The Belgian Minister of Maggie De Block called the attitude of Novartis company that owns AveXis company (producer of Zolgensma) as cynical “You cannot start asking [for] prices that are not in proportion to the real development cost, just because the therapy happens to be groundbreaking.”

She tried to highlight that by doing that Novartis tries to make the most of it. „…“It’s very cynical to see that some companies consider society as nothing more than a cash cow.” And I personally agree with this statement but despite that I do not think it should be a reason to let suffer those who need this treatment. EMA (European Medicines Agency) is still evaluating the treatment. The Agency is the one that should negotiate and mothers are the ones who have to fight. To fight not only with the serious disease of their beloved children but also to fight with institutions that do not take the responsibility. Responsibility of governments is not only about to highlight if something is too expensive but it is their responsibility to take care that prices of medicaments should have very little impact on common people. Governments and European institutions such as EMA should do everything to make treatment accessible. By the time it happens it is necessary for the governments to cooperate and create suitable conditions to apply the treatment.

Children like Riško and Alex do not have time to wait whether the application of the treatment will be approved or not. Parents of these small children got into very difficult situation when they had to make a decision whether to continue in treatment with Spinraza that slows down the progress of the disease or take the risk and ask the public for help so that to be able to provide Zolgensma for their children.  It was not easy to make this decision. They studied lots of information, communicated with AveXis company and discussed. They also communicated with the hospital in Boston that covered clinical tests for this treatment. After learning about positive experience of children from other EU countries the mothers from Slovakia decided to fight as well. So this is not just a desperate activity of mothers who try to catch at a straw but this is a decision made based on long term acquisition of information.  So why has our government decided not to help? This is not about the financial part of the situation. Unfortunately the parents face another problem as well. When they raise the money (and I really believe that this situation leaves nobody indifferent) they will have to face another problem – where can the treatment be applied?

I have just learnt that the Bethesda Children Hospital in Budapest has decided to help. To Riško, Alex, Alexej and Amélia the treatment can be applied in their hospital. But the subsequent checkups will have to be covered in Slovakia.  Believe or not but it was the Hungarian government that changed its law to enable children from Slovakia to undergo the treatment in Budapest. But it is sad to see that help comes from Hungary while our government shows no engagement.

And for that reason it is just up to us whether we show our power and help these children. Riško needs to collect 1.9 mil. € by March 31, 2020 as two weeks before application of  Zolgensma treatment he will have to undergo some medical tests. Alex needs the same amount by June 30, 2020. Will we become the ones who will not only standby but who will take action so that we can give these little fighters a chance to live? A chance to have a life they deserve? Or will we just stand by and pretend it is none of our business?  Time is merciless and EMA will not approve the treatment for Riško and Alex in time.

Children who have been lucky enough to get Zolgensma prosper and it is clear from many videos, positive reactions from parents or other people.

RIŠKO:

FB: https://www.facebook.com/RiskoSMA1/

IBAN: SK89 0900 0000 0051 6672 6293

For contributions from abroad:

IBAN: SK89 0900 0000 0051 6672 6293

BIC/SWIFT: GIBASKBX

Account name : BOJOVNÍK

Bank name: Slovenská sporitelňa a.s.

Transparent account:

https://www.transparentneucty.sk/#/ucet/SK8909000000005166726293

Pay pal: https://www.paypal.me/RiskoSMA1

https://www.gofundme.com/f/the-little-fighter-with-serious-diagnosis?fbclid=IwAR0aXbszidhlGGkaQGq5rAhQt8OJgBazTPRqAxfBMruaB-A8jAvDWpkwTh8

Ľudia ľuďom (People to People): https://www.ludialudom.sk/vyzvy/7135

BIC/SWIFT:FIOZSKBAXXX
Názov účtu: Alexsma

ALEX:

FB: https://www.facebook.com/Alex-SMA-bojovn%C3%ADk-102602567940372/

IBAN: SK18 8330 00000021 0168 4316

For contributions from abroad:

IBAN: SK18 8330 00000021 0168 4316

BIC/SWIFT: FIOZSKBAXXX

Account name : Alexsma

Bank name: Fio banka a.s.

Transparent account:

https://ib.fio.sk/ib/transparent?a=2101684316

Pay pal: paypal.me/pools/c/8lpXL3fDB9

https://www.gofundme.com/f/alex-with-sma-needs-the-world-most-expensive-drug?utm_source=facebook

Ľudia ľuďom (People to People): https://www.ludialudom.sk/profil/detail/39194

 

You can support them by purchasing these mugs https://nakupto.sk/kategoria-produktu/risko__alex/

RIŠKO:

https://www.facebook.com/RiskoSMA1/videos/590637688443981/

 

ALEX:

Stories of children after drug administration Zolgensma:

 

Pekný deň prajeme:) Dostalo sa nám do rúk video o dievčatku s SMA. Myslím, že mi došli slová. Čo vy na to? Ďakujeme Robo Ščepán za natitulkovanie. :)))

Uverejnil používateľ Lucia Kohaniová Jarušinská Sobota 15. februára 2020

 

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