Question 1

Who may be considered for Kanexthal?

Accepted Answer

Eligibility depends on medical criteria such as diagnosis, transfusion burden, genotype, organ function, and other factors assessed by qualified physicians.

Question 2

Does gene therapy replace routine monitoring?

Accepted Answer

No. Long-term follow-up and periodic monitoring are typically recommended after gene therapy.

Question 3

What are the risks related to conditioning chemotherapy and hospitalization?

Accepted Answer

The main risks are associated with myeloablative conditioning (a chemotherapy process) and include decreased white blood cells, platelets, and neutrophils, along with nausea, vomiting, and mouth sores. Most reactions are manageable, with Grade 1-2 toxicities being the most common.