ISBN-13: 9780367483845 / Angielski / Twarda / 2020 / 146 str.
ISBN-13: 9780367483845 / Angielski / Twarda / 2020 / 146 str.
So far more than 1000 trials are currently ongoing with gene and cell therapies. Cure is no longer a dream for several monogenic disorders. However there is still uncertainty on how those products will be accessed by patients and at what price. This book aim to show how the regulatory environment has evolved to analyze and review those therapies.