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REBLOZYL (Luspatercept) Approved by FDA

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Vikramjit Singh:
Great news!

Andy Battaglia:
Not yet approved for children.

https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/761136lbl.pdf


--- Quote ---8.4 Pediatric Use
Safety and effectiveness in pediatric patients have not been established. Based on findings in
juvenile animals, REBLOZYL is not recommended for use in pediatric patients [see NonClinical Toxicology (13.1)].
--- End quote ---

Andy Battaglia:
Emby,

Reblozyl is an erythroid maturation agent. That means that it takes immature red blood cells that would not normally mature in thalassemics and it causes them to mature, adding to the RBC total and the Hb level. It is given as a subcutaneous injection every few weeks.

smit-17:
How many % reduction  in transfusion burden
For β0/β0- major?

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