Question 1

What types of rare diseases does PTC Therapeutics focus on?

Accepted Answer

PTC Therapeutics pioneers pharmaceutical science to transform lives and treat rare diseases, such as Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Huntington's Disease, and Leukemia.

Question 2

What is the focus of PTC Therapeutics' gene therapy center of excellence?

Accepted Answer

PTC Therapeutics has a Gene Therapy Center of Excellence that focuses on developing innovative therapies to address the underlying cause of rare diseases.

Question 3

What are some of the key therapeutic areas that PTC Therapeutics is involved in?

Accepted Answer

PTC Therapeutics is involved in various therapeutic areas, including metabolism (Familial Chylomicronemia Syndrome, Familial Partial Lipodystrophy, Phenylketonuria), neurology (Amyotrophic Lateral Sclerosis, Aromatic L-Amino Acid Decarboxylase Deficiency, Duchenne Muscular Dystrophy, Friedreich Ataxia, Hereditary Transthyretin Amyloidosis, Huntington's Disease, Spinal Muscular Atrophy), and more.

Question 4

What is the company's mission?

Accepted Answer

PTC Therapeutics' mission is to transform lives and treat rare diseases by harnessing its scientific platforms to create new therapies that address the underlying cause of the disease, and to deliver on its promise to create more moments for people living with rare diseases.

PTC Therapeutics

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Frequently Asked Questions about PTC Therapeutics

What types of rare diseases does PTC Therapeutics focus on?

What is the focus of PTC Therapeutics' gene therapy center of excellence?

What are some of the key therapeutic areas that PTC Therapeutics is involved in?

What is the company's mission?

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