December 20, 2022
Approximately 80% of rare diseases have a genetic etiology. Gene therapies have the potential to transform the treatment of rare diseases and may provide a functional cure for some. However, these…
June 29, 2022
With the increasing number of approved innovative, high-cost gene therapies, value assessment and novel approaches to reimbursement are rapidly gaining importance. To assist developers of gene…
December 15, 2021
Approximately 80% of rare diseases have a genetic etiology. Gene therapies hold the potential to transform the treatment of rare diseases and may provide a functional cure for some. However, these…