Primary Recipient

PTC THERAPEUTICS, INC.

Amount

$996,430.00

Award Date

9/28/2009

Administering Agency

Health and Human Services, Department of / National Institutes of Health

CDFA Number

93.701

CDFA Program Title

Trans-NIH Recovery Act Research Support

Description

Severe hemophilia A and B (HA/HB) are disabling and life-threatening orphan disorders caused by a genetic defect. No treatments to correct this genetic defect are available. In previous studies in animals and humans, ataluren has shown the potential to tr