What is SMA?

“SMA (Spinal Muscular Atrophy) is a disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe. It is the number one genetic cause of death for infants. SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene produces a protein that is critical to the function of the nerves that control our muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating and often fatal muscle weakness.SMA can affect any race or gender. Individuals with SMA have difficulty performing the basic functions of life, like breathing and swallowing. However, SMA does not affect a person’s ability to think, learn, and build relationships with others” (CureSMA.org).

Types Of SMA:

There are four primary types of SMA—I, II, III, and IV—based on the age that symptoms begin:

Type 1: Also known as Werdnig-Hoffmann disease, SMA Type 1 is the most common (60%) and severe form, usually diagnosed during an infant’s first 6 months. Babies with SMA Type 1 face many physical challenges, including muscle weakness and trouble breathing, coughing, and swallowing. They may need breathing assistance or a feeding tube. If not treated, Type 1 can be fatal early on in life.

Type 2: It is usually diagnosed after 6 months of age, but before 2 years of age. The first sign is often a delay in meeting motor milestones or failing to meet milestones entirely. Individuals with SMA Type 2 can typically sit up without help, though they may need assistance getting into a seated position, but they are unable to walk and will require a wheelchair.

Type 3: Also called Kugelberg-Welander disease or juvenile SMA, Type 3 is usually diagnosed after 18 months of age, but before 3 years of age. However, SMA Type 3 can be diagnosed as late as the teenage years. Individuals with SMA Type 3 are initially able to walk but have increasingly limited mobility as they grow and eventually, many need to use a wheelchair.

Type 4: SMA Type 4 is very rare, less than 1% of all diagnosed cases. It usually surfaces in adulthood, and it leads to mild motor impairment. While symptoms can begin as early as age 18 years, they usually begin after age 35 years.

Treatment:

Up to a few years ago there was no cure for SMA, Doctors could only provide supportive treatment besides genetic counselling and prenatal diagnosis to avoid another child with the same disease. Now with the advancement of research, three new treatment options have been identified & are too expensive. Except for the compassionate use where few of the patients could get the gene therapy or risdiplam, it is extremely difficult to get these medicines for the SMA community across India.

The U.S. Food and Drug Administration approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA) in May 2019, the most severe form of SMA and a leading genetic cause of infant mortality costing to be 16 Crores (excluding Taxes)

Issue:

Now the issue is the Cost, The Per Capita Income at current prices during 2020-21 is estimated to be ₹ 127,768 (National Statistical Office) & unfortunately due to the Cost factor Parents are desperately trying their best efforts to save their kids via different platforms such as Crowdfunding, Media, Reaching Leaders & influencers etc.,

Noor Fatima Seven-month-old girl from Bikaner, who was suffering from a rare spinal muscular atrophy (SMA) type -1, passed away, they had collected 40Lac Rupees via the Crowdfunding platforms but due to time constraints they could not reach the target.

Why the Parents have to rely every time on the crowdfunding platform where they will be no absolute assurance to reach the target?

Article 21 in The Constitution of India mentions that No person shall be deprived of his life or personal liberty except according to procedure established by law [Protection of life and personal liberty]

Just feel the agony of those parents who’re reaching to every person to procure the essential drug to save their Kids from SMA & are helpless

Via this Petition, we all are requesting to the Government of India to consider the following as soon as possible:

1.     To provide the above medicine at a reasonable price for the Kids considering the socioeconomic factors

2.     To Create a Platform where the People all over the world can deposit directly to the effected people with respect to SMA as per the price considered by Government -------(1)

3.     To consider the SMA expense eligible u/s Sec 135 of the Companies Act,2013 & make it a mandatory to a certain % of funds every year to be deposited via the platform as per -------(2)

4.     Awareness about this disease & measures to be taken considering the time constraints & to have an atleast one authorized hospital in every state of India for the same.

5.     Proper Control over the Crowdfunding platforms as said by the Kerala HC in the recent proceedings (09-07-21)

“Every Life is important”

“Alone We can do so little, But together we can do so much”-Helen Keller ; it’s not just the Government We as the People have to come forward as well & do our best in helping these kids to lead a happy, healthy & successful life.

(Attached Image Source : Instagram, Only for Reference)