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 AADC Deficiency

About Angelman Syndrome

About Duchenne Muscular Dystrophy (DMD)

About Familial Chylomicronemia Syndrome (FCS)

About Friedreich’s Ataxia

About hereditary transthyretin (hATTR) amyloidosis

About Spinal Muscular Atrophy (SMA)

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