Improving Rare Disease Awareness: Bruce Dezube, MD
The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.
“Rare disease brings together government agencies, including both the funding and the regulatory agencies, pharmaceutical industries, and patient advocates. And, their priorities can sometimes differ... These different groups are involved with every aspect [of therapy development] and every aspect is working, but every aspect could be working even better.”
Monday, February 28, marked international Rare Disease Day, which recognizes over 7000 rare conditions that affect over 300 million people worldwide. The day brought together researchers, patients, advocates, and policy makers in conversations on how to improve awareness and management of rare diseases.
Severe combined immunodeficiency (SCID), glioblastoma, and chronic lymphocytic leukemia (CLL) are 3 rare diseases that companies such as Mustang Bio are developing therapies for. These programs, lentiviral gene therapies in SCID and glioblastoma and chimeric antigen receptor (CAR) T-cell therapies in CLL, are in phase 1 studies with planned phase 2 parts.
CGTLive spoke with Bruce Dezube, MD, senior vice president and head, clinical development, Mustang Bio, to learn more about the importance of Rare Disease Day as well as the role of collaboration and advocacy to improve rare disease awareness and therapy development.
Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
April 20th 2024Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
