Welcome to HemDifferently by BioMarin

A Healthcare Providers’ resource for exploring gene therapy research

It’s important for all members of the community to understand what makes gene therapy research unique from other types of research, from the research process and technology, to the technical and practical requirements. We created HemDifferently as an educational platform to help guide conversations both internally among institutional stakeholders and externally with patients and their family members.

Institutional Considerations for Investigational Gene Therapy

Today, many gene therapies are being studied in people to determine if they are safe and effective. The ongoing research may present different requirements for healthcare providers and patients. Below are some potential practical considerations for addressing as your institution considers gene therapy.

Each gene therapy will be evaluated by the FDA upon submission. No gene therapy for hemophilia A or B has been determined to be safe or effective or approved for use.

Site readiness

Infrastructure, training, business, and administrative considerations

Patient conversations

Setting appropriate patient expectations

Patient identification

Determining a patient's candidacy for different gene therapies

Insurance process

Understanding access considerations

Dosage and administration

Protocols to administer gene therapies

Patient monitoring

Short- and long-term follow-up