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SMARD

(Spinal muscular atrophy with respiratory distress type 1)

is a nerve disease caused by a very rare genetic defect. It causes infants or toddlers to lose control of their muscles, ultimately suffering severe respiratory distress due to the paralysis of their diaphragm. Children usually only have a very short lifespan between six weeks and a few years. Until now, there is no cure for this disease.

Is there any hope?

Yes, there is! A team of researchers in the United States is working on a very promising project to develop a gene therapy. A subsequent study could already start next year and perhaps bring salvation for the affected children.

Can I help?

Yes, you can! A lot of financial support is required to push this study through the various research steps. Currently, the research team requires funding of 2.1 million US-dollars to bring the study to the next stage. Any donation helps to win this fight against time.

stories of SMARD-kids

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Little Jule was born in Hamburg, Germany, and just celebrated her sixth birthday.

She has been suffering from SMARD all her life. The disease is already

paralysing her muscles. Mudi is one year old and since the beginning of his tiny life suffers severly. After times of despair, there is finally a glimmer

of hope for these kids and their families.

Together with SMASHSMARD USA (www.smashsmard.org), we are committed to promote the study on combating SMARD.

There are now only approx. 250.000 USD missing.

Become a donor today!  

We will happily provide you with a tax receipt.

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SMASHSMARD Deutschland e.V.

IBAN: DE37 2005 0550 1500 3591 85

BIC: HASPDEHHXXX

This study would not only be a godsend for these kids and all others suffering from SMARD. Furthermore, it would be a giant leap for gene therapy as a whole and could also impact the cure of other genetic defects.

View more information about SMARD

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SMARD is a very rare congenital defect on the IGHMBP2 gene. This genetic defect leads to the nerve disease called Spinal Muscular Atrophy with Respiratory Distress, where an important protein is not formed correctly, which causes nerve cells not to be properly supplied. Impulses from the brain do not sufficiently reach the muscles throughout the body which causes the muscles to retract. This also affects the diaphragm and therefore the ability to breathe.