Risdiplam showed medically-meaningful and statistically significant results patients with type 2 and type 3 SMA.
Autologous gene therapy for rare diseases including primary immune deficiencies, inherited metabolic disorders and blood disorders (Image: Orchard Therapeutics).
Deal includes $745 million in potential additional milestone payments based on success of Agilis' central nervous system therapies.
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PTC Therapeutics Inc is a global biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, small-molecule therapeutics. Its target area is RNA biology post-transcriptional control processes of the events occurring in a cell when a RNA messenger is copied from DNA during the transcription process. The discovered products address multiple therapeutic areas, including rare disorders, such as nonsense mutations in Duchenne muscular dystrophy and oncology.
| Website: | www.ptcbio.com |
| Email: | akane@ptcbio.com |
| Main Phone: | +1 908 222-7000 |
| Address: | 100 Corporate Court |
| State: | NJ |
| City / Town: | South Plainfield |
| Country: | US |
| Postal Code: | 07080 |
| Exchange: | NGS |
| CEO: | Stuart W. Peltz |
| Employees: | 967 |
| NAICS: | Pharmaceutical Preparation Manufacturing(325412) |
Risdiplam showed medically-meaningful and statistically significant results patients with type 2 and type 3 SMA.
Autologous gene therapy for rare diseases including primary immune deficiencies, inherited metabolic disorders and blood disorders (Image: Orchard Therapeutics).
Deal includes $745 million in potential additional milestone payments based on success of Agilis' central nervous system therapies.
Here's a look at an upcoming catalyst for PTC therapeutics.
Aging baby boomers face increased cancer risk, and growing numbers of obese and overweight Americans face metabolic disorders that could lead to cardiovascular disease. Debjit Chattopadhyay of
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