The approval is grounded in data from a Phase III randomized, double-blind, placebo-controlled, crossover study in NPC patients
The system is intended for some of the tiniest patients—premature infants weighing as little as two pounds
It's a development that marks a potential breakthrough for people living with the rare and debilitating disease Duchenne muscular dystrophy (DMD)
Itvisma is a one-time, fixed-dose therapy designed to tackle the genetic root cause of SMA
The therapy -- Ryoncil (remestemcel-L-rknd) -- will be tested in adults with severe steroid-refractory acute graft-versus-host disease
Pete Pharma will leverage FABRX’s extensive foundation of scientific validation to deliver a commercially viable, pharmacy-ready solution in the US
Vonvendi is the only recombinant Von Willebrand factor replacement therapy with approved indications in adults and children with VWD
Genomics is transforming the future of modern medicine and treatment modalities across specialties
Dr. Prabhavathi brings over 14 years of unparalleled experience in pediatrics and a specialized focus on neurodevelopmental disorders
He is an experienced professional in the pharmaceutical industry with about 30 years of progressive experience
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