- Funding supports continued focus on rare disorders -
SOUTH PLAINFIELD, N.J., June 15 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced the receipt of two government grants to support clinical development programs in rare disorders. The U.S. Department of Defense Neurofibromatosis Research Program has awarded an $822,345 grant to support a new open-label Phase 2 clinical trial of PTC's product candidate PTC299 in neurofibromatosis type 2 (NF2), a rare genetic tumor. Separately, the Food and Drug Administration (FDA) Office of Orphan Products Development has awarded PTC a four-year, $1.6 million grant towards its ongoing pivotal trial of ataluren (PTC124(TM)) in nonsense mutation Duchenne and Becker muscular dystrophy (nmDMD/BMD).
"We are honored to receive these grant awards from the FDA and from the Department of Defense," said Stuart W. Peltz, Ph.D., president and Chief Executive Officer of PTC Therapeutics. "Obtaining peer-reviewed grant awards are a strong endorsement of PTC's science and support our strong commitment to developing innovative medicines in areas of high unmet medical need."
Patients with NF2, a rare genetic disorder of the central nervous system, typically develop tumors that compress the auditory nerves, often leading to deafness and problems with balance. If unchecked, NF2-related tumors may eventually damage other cranial nerves and the brainstem, becoming life threatening. Current standard treatments for NF2-related tumors include surgery and radiation therapy. VEGF plays a critical role in angiogenesis, or the formation of new blood vessels, and supports tumor growth in patients with NF2. PTC299 is a novel, orally administ
|SOURCE PTC Therapeutics, Inc.|
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