A drug from Biogen (BIIB) on Friday became the first approved therapy for a rare and devastating genetic disease, giving hope to patients and marking a major milestone for a biotech giant at a crossroads.

The therapy, Spinraza, is meant to treat an inherited disorder called spinal muscular atrophy by tinkering with a defective gene. Children born with the disease don’t make enough of a protein called SMN, which is key to motor development. Spinraza works by amplifying the gene responsible for SMN, thereby boosting its production. The FDA approved the drug to treat both children and adults.

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  • Hello, I am living in Turkey, a womanizer Text. And I have a child with SMA type 1 patients.My child is 5 months old, I’m asking you to help me. You produce the “Spinoza” (nusiner if I want to get information from you about a drug named.Please help me.People with this condition in the whole world you are the hope of.What is the price of this medication ? I’d appreciate it if you notify me with an email. I wish successful work, thanks

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