Pharmaceutical Technology

Rocket’s gene therapy Kresladi wins FDA approval in rare immune disease

Upon approval, Rocket has been awarded a priority review voucher, which currently fetch around $200m on the secondary market.

Rocket Pharma shares climb as FDA approves first therapy for deadly childhood disorder

March 27 (Reuters) - Rocket Pharmaceuticals shares jumped 10% on Friday before the bell after the U.S. Food and Drug Administration approved its gene therapy for a rare and often fatal immune disorder ...

Rocket Pharma rises as FDA approves gene therapy

Rocket Pharmaceuticals (RCKT) stock jumps as FDA approves its gene therapy Kresladi for a rare immune disease called ...
Nature

Human CD33 deficiency is associated with mild alteration of circulating white blood cell counts.

The Nature Index 2025 Research Leaders — previously known as Annual Tables — reveal the leading institutions and countries/territories in the natural and health sciences, according to their output in ...
Scientific Research Publishing

Deficiency of Factor V of Coagulation: About a Case ()

Context: Factor V deficiency is a rare abnormality of coagulation as para-haemopilia, originally described by Owren in 1947, ...