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We’re seeking a cure for a rare disease –
Big Hearts wanted!

SMASHSMARD e.V. is an initiative to support medical research and awareness for the rare disease SMARD. Our aim is to make a cure a reality in order to give back a quality of life to the affected children.

Donate now!

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 this 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 dollars to bring the study to the next stage. Any donation helps to win this fight against time.

Jule’s story

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

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

paralysing her muscles. After years of despair, there is finally a glimmer

of hope for Jule and her family.

Together with SMASHSMARD USA (www.smashsmard.org), we are committed to promote the study on combating SMARD. More than 700,000 USD have already

been donated. Unfortunately this is not yet enough.  Become a donor today!  

We will happily provide you with a tax receipt.

SMASHSMARD Deutschland e.V.

IBAN: DE37 2005 0550 1500 3591 85

BIC: HASPDEHHXXX

 

This study would not only be a godsend for Jule and the other kids 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.

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SMASHSMARD Deutschland e.V.

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View more information about SMARD

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.