Help Ayah fight
SMA

Behind the beautiful smile is a courageous 14-month old girl fighting SMA, a gene defect. It results in a loss of muscle mass that affects both motoric functions and internal organs.

Luckily, there is now a new treatment that can stop the degeneration of Ayah’s muscles. Zolgensma is a brand new gene therapy that can repair her gene defect – a one-time treatment effective for children below the age of two years. Ayah turns two in January 2022.

Zolgensma is approved in EU by the European Medical Agency (EMA) but the Danish Medical Council is yet to recommend it as a standard treatment in the Danish hospitals for SMA type 2, and therefore Ayah cannot receive the treatment in Denmark. For this, she needs to raise the hefty amount of 2.4 million USD to travel abroad for treatment.

All donations go entirely to Ayah through the Ayah Mithika Lundt Foundation.

Ayah
What is SMA?

According to the Muscular Dystrophy Association National Office, Spinal muscular atrophy (SMA) is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscle).

Persons suffering from SMA lack a vital gene that produces a protein necessary for motor neurons’ functioning, which is in charge of transmitting impulses to the muscles.

Without this protein, motor neurons lose their function and die out. Consequently, without the impulses from the motor neurons, the muscles undergo progressive weakening and atrophy.

The atrophy starts with large muscle groups, arms, legs and neck. After some time, it progresses to smaller muscle groups in charge of vital functions of swallowing, breathing, or coughing, eventually leading to death.

FAQ

According to the Muscular Dystrophy Association National Office, Spinal muscular atrophy.
(SMA) is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscle). 

The human body is composed of myriad cells and genes that make us who we are. A mutation in the SMN1 gene causes SMA.

SMN is an acronym that stands for Survival Motor Neuron. Everyone has a copy of the SMN1 gene, and it’s known as the SMN2 gene. This SMN2 gene produces the same protein type that the SMN1 gene has, only at lower amounts, as explained in the SMA news today website.

There are currently two drugs approved for the treatment of SMA

Spinraza
Fortunately, Ayah is currently on Spiranza, which works by trying to repair the SMN2 gene. Even though the treatment improves the SMN2 gene, it still produces a less functional protein that the SMN1 gene.

Spinraza is a lifetime, invasive treatment. Ayah receives the medicine through a spinal cord needle, a lumbar puncture procedure, or a spinal tap.

Risdiplam
Risdiplam is currently only approved in the USA. Like Spinraza, the treatment also targets on improving SMN2 gene.

Zolgensma
This Groundbreaking gene therapy medication replaces the missing, defective SMA1 gene instead of working on the weaker SMN2 gene. If administered in good time, Zolgensma provides the patients with fully functional SMN protein.

It is the first gene therapy of its kind, and it is administered only once. Zolgensma is the most expensive treatment globally. It costs 2.1 million USD and is currently approved in a few countries. 

According to Novartis, the drug company behind Zolgensma, the medication with a price tag of 2.1 million for a single treatment ‘dramatically transforms the lives of families affected by this devastating disease.’ The medicine is a one-time-only gene therapy treatment for children aged less than two years - a medical miracle of its kind.

Whether Zolgensma is a cure or not is difficult to answer. Children who receive Zolgensma before their symptoms appear to develop like normal children. So, for these pre-symptomatic children, Zolgensma works like a cure.

After their symptoms appear, children who receive treatment achieve typical developmental milestones. Every child reacts differently to the treatment.

However, in all cases, Zolgensma seems to produce positive results, but in different magnitudes. Therefore, we like to call Zolgensma an “almost cure”.

Zolgensma is approved in EU by the European Medical Agency (EMA) but the Danish Medical Council is yet to recommend it as a standard treatment in Danish hospitals, and therefore Ayah cannot receive the treatment in Denmark.

Yes, we do. Denmark has universal health care available to all. However, since Zolgensma isn’t approved in the country, it is up to Ayah’s parents to raise the money and seek treatment abroad while they still can. 

At the age of 14 months, Ayah is still in good spirits despite the disease’s progression. Unfortunately, she can neither sit without support nor fully roll on her side while lying down. She cannot stand or crawl as her legs are not as strong as her arms.

Despite everything, Ayah continues to eats well and breaths well without the aid of a machine help.

With your generous donation, you will help Ayah receive the treatment to replace the genes responsible for muscle gain and prevent her organs from deteriorating. 

There are several ways you can help.

First and foremost, our target is to achieve as many donations as possible to reach the hefty cost of 15 million DKK (2.5 million USD).

All donations go to Ayah Mithika Lundt Foundation.

You can help;
- By sharing this message and help create awareness of the disease.
- By donating on the Go-FundME page
- Raise awareness about SMA and lobbying for relevant authorities to approve Zolgensma in your respective country.

Kindly follow our Facebook page for regular updates and ways to #HelpAyah. 

Sponsors

https://www.pamojakenya.org/
https://studiobornholm.dk/
https://lasertryk.dk/

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