PRODUCT LITERATURE

Haemophilia, state of the art and new therapeutic opportunities, a regulatory perspective

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Haemophilia A and B are rare bleeding disorders. Over the past decades, they have been transformed from debilitating diseases to manageable conditions in the Western world. However, optimizing haemophilia care remains challenging in developing countries. Several challenges and unmet needs remain in the treatment of the haemophilia limiting the QoL of patients. These challenges are now being addressed by extended half-life recombinant factors, rebalancing and substitution therapies. Gene therapy and genome editing show promise for a definite clinical cure. Here, we provide an overview of new therapeutic opportunities for haemophilia and their advances and limitations from a regulatory perspective

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: .,

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Language

English

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NONE

Content Type

LITERATURE

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Subject(s)

Beriate

Specific Detail Info

Br J Clin Pharmacol. 2021;87:4183–4196.

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