Regulatory Agency and Partner Announcements

Biomarin
Biomarin provides updated 4-year data from their Phase 1/2 Valoctocogene Roxaparvovec gene therapy trial in Late-Breaking Oral Presentation at World Federation of Hemophilia Virtual Summit. To read the full press release, please click here.
June 2020


EHC-EAHAD Joint position statement on Gene Therapy
May 2020