Give Michael a life

Michael is fighting Spinal Muscle Atrophy SMA Type 1 – most children with this disease do not survive more than two years. Every day a little piece of him is irreversibly lost. 

A lifesaving gene therapy is currently only available in the US. $ 2.1 Million – it is an overwhelming amount. We tried everything. This is our last chance to safe his life – if we act fast.

A human life is priceless. Together we can give Michael a chance to live!

The novel drug Zolgensma was approved in May only in the US for use in children under two years old. In Germany, it is still pending and there is no prospect of early treatment. We tried everything. 

Fate puts a hard test on us. The probability of SMA of newborns is 1:10000.  

Time is running out for Michael as his motoneurons decay irretrievably every day. The gene therapy would save his life. Only a rapid treatment can be successful because it builds on what is left in his body. 

 

A child – the greatest gift. 

Michael was born on the 15th of August 2018 as a healthy, happy baby. For us as a family he was the greatest gift – his big sister was hoping for a sibling for so long. But when he was six weeks old, we noticed that was dropping his arms abruptly and looked very powerless. A few days after that, the frightening diagnosis was made in the Stuttgart Olgahospital.

We started the only available therapy immediately: unfortunately, it doesn’t show very much impact, Michael is struggling with increasing respiratory problems on a daily basis. The only therapy available is an injection in the spinal cord under general anesthesia every four months. 

The drug approved in the US is a single dose: after one infusion, Michael has a chance to live and develop, play, laugh, even walk like other kids…

SMA is a rare, genetic disease that leads to progressive muscle weakness, until the breathing stops…

Spinal Muscular Atrophy is a life-threatening disease caused by a defective or missing SMN1 gene. Without this gene, infants with SMA lose the motoneurons responsible for muscle functions such as breathing, swallowing, speaking, and walking.  

Left untreated, muscles become weaker progressively, this eventually leads to paralysis and ultimately permanent ventilation or death by age two in more than 90% of the cases. 

Zolgensma is the first and only gene therapy approved for the treatment of SMA.

Zolgensma has the potential to make a truly transformative impact on this life-threatening disease. It is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous infusion. 

“A diagnosis of SMA is devastating, leaving untreated babies who have the most severe form Type 1 with painfully short, highly medicalized lives, during which they are unable to lift their heads, sit or roll, have difficulty swallowing and breathing and need 24-hour care. In the clinical trial we conducted with Zolgensma, all children were alive at the conclusion of the study and many were able to sit, roll, crawl, play and some could walk. This level of efficacy, delivered as a single, one-time therapy, is truly remarkable and provides a level of unprecedented hope for families battling SMA Type 1. We now have data four years out from the trial, and we see the durability of this gene therapy.”

Doctor Mendell can treat Michael immediately, as soon as we can afford the therapy. Every donation counts! It’s a huge sum, but not impossible if a lot of dear people help out. For Michael. For one life. We need YOU and all your friends. Together we can SAVE Michael! 

Help for Michael

Our fundraising is possible due to the generous support of the Deutschen Muskelstiftung (Muscle Foundation) and the Initiative – Hilfe für Einzelschicksale e.V (Initiative Help for individual Fates). We’re grateful for their support.  

Please support us! Every contribution counts. Share our story with friends and family and help us raise the necessary funds for the drug.

If Zolgensma is available in Europe on time and if the drug is covered by health insurance, we will use every single cent to help Michael live a dignified life and support other families affected by SMA Type 1, 2 or 3. 

Bank Transfer:  Please do not forget the reference “Michael” 

Deutsche Muskelstiftung 
Bank fuer Sozialwirtschaft (Karlsruhe, Germany)

IBAN: DE11 6602 0500 0008 7390 00 

Reference (wire transfer form): Michael 

SWIFT/BIC: BFSWDE33KRL

Credit Card:

RETTE MICHAEL
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PayPal Payments (collected by Initiative Hilfe für Einzelschicksale e.V.)

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2 000 000 thanks for YOUR support!


Contact

Parents of Michael: Marina und Oleg

MichaelinNot@gmail.com

TEAM MICHAEL

We are Michael’s family and friends. Together with YOU, we want to give him a chance to live and lead a normal life. Every action counts! THANKS for YOUR support!

DRAWINGS

Michael’s big sister drew a flower meadow on which they can dance and laugh together. She loves to sing to him… “It makes him smile so happily!”