Dear Duchenne Community, As you might have seen, the Office of Drug Evaluation I of the FDA provided a Complete Response Letter (CRL) for the application to market ataluren in […]
PTC Therapeutics received a Complete Response Letter from the Food and Drug Administration (FDA) for Ataluren’s New Drug Application (NDA). This means that the FDA has completed its review of […]
Dear Duchenne Community, On behalf of all of PTC, we want to thank all of you for supporting the advisory committee review of ataluren. As you know, the meeting was […]
Dr. Kevin Flanigan from Nationwide Children’s Hospital provided an update on his Dup2 research during a CureDuchenne hosted call with families on September 6. Back in 2010, Dr. Flanigan told […]
NEWPORT BEACH, Calif., June 15, 2017 – RASRx announced today that the United States Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) for its compound RASRx1902 […]
CureDuchenne is pleased to see that the FDA will hold an Advisory Committee Meeting (AdCom) for PTC Therapeutics’s drug Ataluren. We are hopeful for a full and fair review which […]
April 20, 2017 Dear Duchenne Community, Today we announced that we have completed our acquisition of Emflaza™ (deflazacort), the first FDA-approved, anti-inflammatory therapy for Duchenne Muscular Dystrophy (DMD) patients […]
ROCKVILLE, Maryland, March 24, 2017 ReveraGen BioPharma Inc, a privately held corporation, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for vamorolone (VBP15) […]
CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community Dear Members of the Duchenne Community: […]
