)
)
Addressing Challenges of Advanced Therapies of Rare Diseases
-
Challenges of bringing advanced therapies to patients with rare diseases.
-
Efforts to improve how academic developers can bring advanced therapies into the clinic.
-
Successes and market failures within the European landscape.
-
Advances in the field, especially in vivo approaches as potential game changers.
-
Examples of emerging solutions (e.g., new payment models, patient-driven research, regulatory initiatives).
