[Update: August 17]
Thanks to the generosity of the donors, $2.9 million has been raised in just 10 days to treat little Devdan with the world’s most expensive drug. According to the ST report, all the collected money will go towards buying the Zolgensma drug to treat the 22-month’s old neuromuscular disorder.
Devdan’s parents are running a campaign in association with Ray of Hope to collect around $2.868 million for his treatment.
August 5
It’s extremely traumatic to see your child suffer in front of your eyes every day. Rare genetic disorders can be hard to detect and parents often get caught up in a complex web of hospital rooms and medicines to keep their little one healthy.
Sadly that’s the plight of little Devdan’s parents today.
The toddler was born with a rare disease called Type 2 Spinal Muscular Atrophy (SMA). This deadly genetic illness can slowly damage the nerve cells in the brain and even the spinal cord.
Spinal muscular atrophy causes extreme weakness in the muscles. As a result, one finds it difficult to do basic things like walking, sitting, talking and even breathing, without help.
Singapore Parents Run Funding Campaign For Their Child Suffering From SMA
Image courtesy: Ray of Hope
The child will turn two this October and can so far “play independently.” However, he requires immediate treatment to stop the progression of SMA and help him survive.
His parents, Dave and Shuwen have urged Singaporeans to come forward and help them raise the money required for Devdan’s treatment so he can be saved in time.
Why SMA Is Dangerous?
SMA most often affects babies and children and makes it hard for them to use their muscles. When your child has SMA, there’s a breakdown of nerve cells in the brain and spinal cords.
Their brain stops sending signals that control muscle movement.
As a result, your child develops weak muscles and can face various physical problems. For instance, trouble controlling their head movement, sitting without assistance and even walking. In severe cases, it can lead to trouble while breathing as well.
4 Primary Types Of SMA
There are four primary types of SMA that affect young children. Devdan is suffering from Type 2.
- Type 1: It is the more severe form. Almost 60 per cent of people suffering from SMA have Type 1. Symptoms appear mostly at birth or within the first six months of life. These kids usually don’t meet the typical milestones like holding their heads up or even sitting. Sadly, most kids with Type 1 SMA die before their second birthday.
- Type 2: Symptoms of this SMA appear when the child is between six months and 18-months-old. They tend to affect the lower limbs. So a child may be able to sit up but they can’t walk. Most children with Type 2 SMA live into adulthood.
- Type 3: This is the mild form of SMA. However, the symptoms of Type 3 SMA appear after the child’s first 18 months of life. In fact, there are some people with Type 3 who don’t have signs of the disease until early adulthood. Symptoms of this type include mild muscle weakness, difficulty in walking and other frequent respiratory infections. These symptoms can also affect the ability to walk or stand. This type of SMA doesn’t significantly shorten life expectancy.
- Type 4: This is a rare adult form of SMA. It doesn’t typically appear until the mid-30s. Muscle weakness symptoms gradually progress, so most people with Type 4 live full lives and also remain mobile.
Spinal Muscular Atrophy Causes
Image courtesy: iStock
Most types of SMAs are caused by a problem with a gene called the SMN1 gene. This gene doesn’t make enough protein required for the motor neurons to work normally.
As a result, the motor neurons break down and they can’t send signals to the muscles.
A child suffering from SMA gets one copy of the SMN1 gene from each parent. On the other hand, if a child gets the SMN1 gene from only one parent, they may not show any signs of SMA. However, there is a risk that they may pass the gene to their kids.
Genetic testing of people with SMA and their parents can help to determine how likely it is for them to have a child with SMA.
SMA Treatment In Singapore
Unfortunately, there’s no cure, but treatment can help to improve the symptoms and also let your child live longer.
So, Devdan’s parents are collecting funds to meet the expensive treatment of buying the gene therapy drug – Zolgensma.
It is a one-time gene therapy treatment for children under two years of age with SMA. It targets the genetic root cause of SMA, by replacing the function of the nonworking SMN1 gene.
The cost of the treatment in Singapore is $2.868 million and so Dave and Shuwen are working with Ray of Hope, which is a crowdfunding charity group to collect funds for his treatment.
According to the latest update (August 4), about $214,000 has been raised so far.
With the help of this treatment, Dave and Shuwen hope their child will manage to improve his muscle strength and it will increase his chance of survival.
If you also wish to help, you can donate here.
News Source: WebMD, Ray of Hope
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