A genetic disorder is an illness caused by abnormalities known as mutations in genes or chromosomes. Most genetic disorders are quite rare and affect one person in every several thousands or millions. At PTC, we are committed to discovering and developing therapeutics for patients living with genetic disorders.

To learn more about our development programs click here.

About Duchenne Muscular Dystrophy (DMD)

About Mucopolysaccharidosis type I (MPS I)

About Spinal Muscular Atrophy (SMA)

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