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Expert Interview

Slingshot members are talking to an expert! The topic is:

Discussing BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec-rvox)for treating Severe Hemophilia A

Ticker(s): BMRN

Who's the expert?

A hematologist with experience in treating Hemophilia A.

Interview Questions
Q1.

How does Valoctocogene Roxaparvovec (BMN 270) work in treating hemophilia A?

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Q2.

What are the main benefits and potential risks associated with Valoctocogene Roxaparvovec as a treatment for hemophilia A?

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Q3.

How does Valoctocogene Roxaparvovec differ from traditional treatments for hemophilia A, such as factor replacement therapy?

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Q4.

Are there any specific patient populations for whom Valoctocogene Roxaparvovec could be recommended or not recommended?

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Q5.

What chances does Valoctocogene Roxaparvovec have to completely eliminate the need for other treatments, such as factor replacement therapy, in individuals with hemophilia A?

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Q6.

What are the potential side effects or adverse reactions associated with Valoctocogene Roxaparvovec, and how are they managed?

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Q7.

What is the long-term safety profile of Valoctocogene Roxaparvovec? Are there any known risks or concerns associated with its prolonged use?

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Q8.

How does the cost of Valoctocogene Roxaparvovec compare to other hemophilia A treatments, and what options are available for financial assistance or insurance coverage?

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Q9.

What is the recommended monitoring protocol for patients receiving Valoctocogene Roxaparvovec? How frequently should patients have follow-up visits or lab tests?

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Q10.

What are the potential future developments or advancements expected in the field of gene therapy for hemophilia A, and how might they impact the use of Valoctocogene Roxaparvovec in the future?

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