Saving little Masha

For our daughter’s first birthday, we are trying to help save the life of little Masha, a girl just her age.

Masha suffers from spinal muscular atrophy (SMA) type 1, a rare genetic disease.

The equation is quite simple:

If her parents can raise the money to pay for a gene-therapy injection, in the next 6 months, she will live a perfectly normal life.

If they don’t, she will leave this world the same she came in it: in pain and unable to even hug her parents.

They have about six more months of her undergoing painful treatments to try and make sure she survives long enough to get that injection, but without this miracle injection, she will die.

After six months of relentless fundraising, they managed to raise only 13% of the injection price. They only have a few more months to raise the remaining 87%.

If they can't do it, they will see their daughter die.

They will then live their whole life knowing they could have saved her, if only they could have raised the money in time.

The question is not about helping her getting temporarily better, or prolonging her life with a severe handicap, or trying some risky procedure with only little chances of success.

No, this is gene editing. The injection exists. It works. The success rate is not 90%. It is not 99%. It is 100%. If you have the money for it, your child lives; if you don’t, she dies.

Now, as inhuman as this bargaining of a child life sounds, it still makes sense from an economics point of view. Such treatments cost billions to develop. If private pharma companies could not recover the investment, they would not even work on it in the first place. The treatment wouldn’t exist. Period.

But, as parents, we simply can’t imagine ourselves in the shoes of his unfortunate little girl’s parents.

Knowing the solution is a simple as one injection, while at the same time knowing you can’t give her that life-saving remedy unless you can find the money for it.

And being given only six months.

The price tag for this single life-saving injection is 2 million dollars. It sounds like an insane amount, but it is not.

We are talking about buying this little girl 75 years of life expectancy.

75 times the amount of life she has had so far.

Some parents do manage to raise the money in time to save their child’s life. They can’t give more than everything they have to save her (and they obviously do). Beyond that already insane price tag, Masha’s parents have to face the extra challenge of trying to raise this amount in rubles, which has depreciated to 0.01 dollar following US sanctions in the past few years. Needless to say, they had little involvement in any potential root causes of those sanctions.

The remainder of the fundraise is just a few thousands of us giving 100 pounds/euros/dollars (instead of buying a new gadget we know we’ll never use more than twice). There is no middle ground: if the parents can’t raise those 2 million before Masha dies, she will remain dead forever.

We’d like to make sure that she is part of those lucky few whose life does not end before that race to pay for the injection is up. Her parents can't raise the required 10k+ per day on their own.

If everyone who reads my content shared to 9 other people and all of us sacrificed 25 pounds (5-10 coffees, or 20 boxes of tissues like the one I just emptied while writing this), we could buy that baby girl’s life right now and give it back to her and her parents. A life.

At her age, Masha should be jumping around, trying to walk and talk, grabbing everything that comes her way. Instead, she can barely move. She can’t even hug her parents.

From experience, this little girl is perfectly aware something is not right with her. She cannot understand why her parents are not helping her get better. We hope she can live to later understand how amazing and beautiful this world has become, with gene-therapy magically able to save the life of unfortunate children like her.

In case Masha’s life was to end before her parents could pay for the gene therapy, they would give the proceed to another cause. But it would not help Masha.

If you usually enjoy my regular free content, please consider donating to save Masha's life.

Any help (besides donating yourself) would also be a blessing: sharing with family, friends and social networks, sharing across company or university networks, volunteering to raise awareness.

The parents are running a campaign in Russia, and my wife is driving a campaign for them in the UK. Neither is currently going fast enough that hope would be very high for Masha.

I can put you in touch with my wife if you want to get involved in any way.