Spinal muscular atrophy is a genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles). It is caused by a loss of specialized nerve cells, called motor neurons that control muscle movement.

1. Spinal
Muscular
Atrophy
(SMA)
Market Insights, Epidemiology
and Market Forecast- 2028

2. Spinal Muscular Atrophy
(SMA)
Spinal muscular atrophy is a
genetic disorder characterized
by weakness and wasting
(atrophy) in muscles used for
movement (skeletal muscles). It
is caused by a loss of
specialized nerve cells, called
motor neurons that control
muscle movement.
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SMA is a motor neuron
disease caused by Bi-allelic
mutation of survival motor
neuron gene 1 (SMN1). There
are two identical genes SMN1
(telomeric copy) and SMN2
(centromeric copy) present at
human chromosome 5q13.
These genes are responsible
for synthesizing survival motor
neuron protein, which is
necessary for the normal
function of motor neurons
that control our muscles.
03
There are many types of
spinal muscular atrophy (SMA
Type 0, I, II, III and IV) that are
caused by changes in the
same genes. The types differ
in age of onset and severity
of muscle weakness;
however, there is overlap
between the types.
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The weakness tends to be more
severe in the muscles that are
close to the center of the body
(proximal) compared to
muscles away from the body's
center (distal). The muscle
weakness usually worsens with
age.
SMA is the most common
genetic disease resulting in
death in infancy and therapies
that improve neuromuscular
function as well as maintain
lifelong general health of
people living are a major
priority and are an unmet
clinical need.
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3. SMN1 is considered the active gene for
survival of motor neuron protein
production, and more than 98% of
patients with spinal muscular atrophy
have an abnormality in both SMN1
genes, which can be caused by a
deletion (95%) of exon 7 or other
mutation.
Survival motor neuron gene 1 (SMN1)
Total prevalent population
EU-5 Countries Prevalence
US Prevalence
SMA Epidemiology
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Spinal Muscular Atrophy total prevalent
population was 19,343 in 7 major
markets (7MM)* in 2017.
*7MM includes:- United States; EU 5 (Germany, France, Spain, Italy, United Kingdom); Japan.
Among the EU5 countries, Germany had
the highest Spinal Muscular Atrophy
prevalent population with 1,643 cases in
2017 followed by UK and France. On the
other hand, Spain had the lowest Spinal
Muscular Atrophy prevalence with 468
cases in 2017.
Spinal Muscular Atrophy total prevalent
population was found to be 13,036 in
the United States in 2017.

4. Spinal Muscular Atrophy diagnosed prevalent population in 7MM is expected to increase at a
CAGR of 0.88% for the study period i.e. 2017-2028.
SMA Market Size
SMA US Market Size
SMA Market Outlook
SMA in 7MM*
SMA Prevalence
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Spinal Muscular Atrophy total US market size was USD 892.37 Million in 2017.
Spinal Muscular Atrophy market size was 1,317 Million in seven major markets in 2017 and is
increasing at a CAGR of 10.42% for the study period (2017-2028).
Spinal Muscular Atrophy total prevalence in the US is expected to increase at a CAGR of 0.77%.
*7MM includes:- United States; EU 5 (Germany, France, Spain, Italy, United Kingdom); Japan.

5. 2010 2011 2012 2013 2014
Drug- ZOLGENSMA
Company- NOVARTIS
SMA Emerging Drugs and Companies
Drug- RISDIPLAM
Company- ROCHE
Drug- RELDESEMTIV
Company- ASTELLAS
PHARMA
Drug- AMIFAMPRIDINE PHOSPHATE
Company- CATALYST PHARMACEUTICALS
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Spinal Muscular Atrophy Market Insights, Epidemiology & Market Forecast-
2028
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