Pompe Disease Therapeutic Market

Pompe Disease Therapeutic Market INDUSTRY INSIGHTS, TRENDS, OUTLOOK, AND
OPPORTUNITY ANALYSIS, 2016–2024
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REPORT DESCRIPTION
Pompe Disease Therapeutic Market : Overview
Pompe disease is an inherited Lysosomal Storage Disorder (LSD) caused by a
deficiency of enzyme acid named alpha-glucosidase (GAA). This causes the
glycogen to accumulate in different kinds of tissues, primarily into smooth muscles,
skeletal muscles and cardiac muscle. It leads to muscle weakness, respiratory
problems, and may also affects the liver and heart. Pompe disease is inherited as an
autosomal recessive genetic trait. There are three major types of pompe disease,
which differ in severity and appear at different age, these types are known as classic
infantile-onset, which starts within a few months of birth, non-classic infantileonset Pompe disease- usually appears by age 1, and late-onset type of Pompe
disease that may shows the symptoms later in childhood, adolescence, or adulthood.
Pompe disease therapeutic market can exhibit immense growth potential due to
presence of limited number of manufacturers, which can significantly enhance and
expand their business potential and scope in this market.
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REPORT DESCRIPTION
Pompe Disease Therapeutic Market : Taxonomy
By Therapy Type:
•
Gene Therapy
•
Enzyme Replacement Therapy (ERT)
•
Chaperone-Advanced Replacement Therapy (CHART)
By Administrational Route:
•
Oral
•
Parenteral
By Patient Type:
•
Infants
•
Adults
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REPORT DESCRIPTION
Pompe Disease Therapeutic Market : Key Players
●
●
●
●
Amicus Therapeutics, Inc.
Valerion Therapeutics, LLC
Sanofi S.A., Oxyrane
Sangamo Therapeutics, Inc.
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Pompe disease is an inherited Lysosomal Storage Disorder (LSD) caused by a deficiency of enzyme acid named alpha-glucosidase (GAA). This causes the glycogen to accumulate in different kinds of tissues, primarily into smooth muscles, skeletal muscles and cardiac muscle.

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