9937 ISTH Gene Therapy Syllabus and Eval Podcast 8 Miesbach

3. What is your role in the care of individuals with hemophilia? Select all that apply *This question is required.

• Physician involved in direct patient care (management and monitoring of bleeding risk)
• Investigator in clinical trials of patients with hemophilia
• Perform assays/assessments in a specialized coagulation laboratory
• Physical, occupational, and/or mental health therapy
• Basic research related to hemophilia, but not related to patient care
• Patient education
• Other, please specify Please enter an 'other' value for this selection.

6. Please rate your ability BEFORE the activity *This question is required.

Space Cell	0 - Not Relevant	1 - Low	2	3	4	5 - High
KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 0 - Not Relevant	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 1 - Low	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 2	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 3	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 4	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 5 - High

7. Copy of Please rate your ability AFTER the activity *This question is required.

Space Cell	0 - Not Relevant	1 - Low	2	3	4	5 - High
KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 0 - Not Relevant	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 1 - Low	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 2	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 3	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 4	KNOWLEDGE of the safety and efficacy of gene therapy for hemophilia based on ongoing clinical trials:: 5 - High

*If yes, or possibly considering changes, please check off what you intend to do differently or incorporate into your clinical practice as a result of this educational activity. Select all that apply *This question is required.

• Obtain additional information on the potential safety concerns associated with gene therapy for hemophilia
• Follow current preclinical and clinical studies utilizing the AAV virus for hemophilia gene therapy
• Discuss safety-related differences between the AAV vector serotypes currently in use for gene therapy with peers and colleagues
• Include safety concerns in discussions of hemophilia gene therapy with patients and caregivers
• Other, please specify Please enter an 'other' value for this selection.