Understanding patient and parent/caregiver perceptions on gene therapy in gaucher disease: an international survey

HIGHLIGHTS

  • who: Tanya Collin-Histed from the International Gaucher Alliance, Silver St, Dursley , ND, UK have published the Article: Understanding patient and parent/caregiver perceptions on gene therapy in Gaucher disease: an international survey, in the Journal: (JOURNAL)
  • what: The survey aimed to benchmark understanding of the educational needs surrounding gene therapy among the Gaucher disease community. The survey was developed following three patient caregiver focus groups underwent review from members of the IGA for readability accuracy before going live to respondents. The survey assessed the opinions, needs, and wishes of the Gaucher disease community via . . .

     

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