The U.S. Food and Drug Administration (FDA) has approved Bluebird bio’s gene therapy for the treatment of a rare neurological disorder, the company said late on Friday. “SKYSONA is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys” with early, active Cerebral Adrenoleukodystrophy (CALD), the company said in a…
FDA Approves Bluebird Bio’s Gene Therapy for a Rare Neurological Disorder
Bluebird’s $2.8 Million Gene Therapy Becomes Most Expensive Drug After US Approval
The U.S. Food and Drug Administration on Wednesday approved bluebird bio’s gene therapy for patients with a rare disorder requiring regular blood transfusions, and the drugmaker priced it at a record $2.8 million. The approval sent the company’s shares 8 percent higher and is for the treatment of beta-thalassemia, which causes an oxygen shortage in…
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