Maddys

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 infantile-onset 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...

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 infantile-onset 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...