Duchenne muscular dystrophy

Target entity: Duchenne muscular dystrophy
Context triple: [PTC Therapeutics, hasDrugIndication, Duchenne muscular dystrophy]

• A. MPS II
  MPS II, also known as Hunter syndrome, is a rare X-linked lysosomal storage disorder caused by iduronate-2-sulfatase deficiency, leading to progressive multi-organ dysfunction and developmental impairment.
• B. spinal muscular atrophy
  Spinal muscular atrophy is a genetic neuromuscular disorder characterized by progressive muscle weakness and atrophy due to degeneration of motor neurons in the spinal cord.
• C. McArdle
  McArdle is a surname most notably associated with Andrea McArdle, the American actress and singer who originated the title role in the Broadway musical "Annie."
• D. Sarepta
  Sarepta is an ancient Phoenician coastal town, known from the Bible as the place where the prophet Elijah stayed with a widow during a severe famine.
• E. Krabbe disease
  Krabbe disease is a rare, inherited neurodegenerative disorder that destroys the protective myelin sheath of nerve cells in the brain and nervous system, typically leading to severe developmental regression and early death in infancy or childhood.
• F. None of above. chosen
• G. Unsure - the case is ambiguous/there is not enough information to decide.

Target entity: Duchenne muscular dystrophy
Target entity description: Duchenne muscular dystrophy is a severe, progressive genetic muscle-wasting disorder primarily affecting boys, caused by mutations in the dystrophin gene and leading to loss of ambulation and life-threatening cardiac and respiratory complications.

• A. MPS II
  MPS II, also known as Hunter syndrome, is a rare X-linked lysosomal storage disorder caused by iduronate-2-sulfatase deficiency, leading to progressive multi-organ dysfunction and developmental impairment.
• B. spinal muscular atrophy
  Spinal muscular atrophy is a genetic neuromuscular disorder characterized by progressive muscle weakness and atrophy due to degeneration of motor neurons in the spinal cord.
• C. McArdle
  McArdle is a surname most notably associated with Andrea McArdle, the American actress and singer who originated the title role in the Broadway musical "Annie."
• D. Sarepta
  Sarepta is an ancient Phoenician coastal town, known from the Bible as the place where the prophet Elijah stayed with a widow during a severe famine.
• E. Krabbe disease
  Krabbe disease is a rare, inherited neurodegenerative disorder that destroys the protective myelin sheath of nerve cells in the brain and nervous system, typically leading to severe developmental regression and early death in infancy or childhood.
• F. None of above. chosen