Research Studies for People with PK Deficiency to Join
Activate-KidsT
Agios Pharmacueticals
Phase 3 clinical trial in children with PKD who are regularly transfused + long term follow up
Activate-Kids
Agios Pharmaceuticals
Phase 3 clinical trial in children with PKD who NOT regularly transfused + long term follow up
RP-L301 Gene Therapy
Rocket Pharmaceuticals
Phase 2 recruitment starting quarter 4 of 2023. RMAT designation from FDA given.