FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation
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The U.S. Food and Drug Administration today granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. It is estimated that about 8 percent of patients with DMD have this mutation.
FDA launches app for health care professionals
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The U.S. Food and Drug Administration announced the global launch of CURE ID, an internet-based repository that will allow the clinical community to report their experiences treating difficult-to-treat infectious diseases with novel uses of existing FDA-approved drugs through a website, a smartphone or other mobile device.
FDA approves first generics of Gilenya
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The U.S. Food and Drug Administration has approved three applications for first generics of Gilenya (fingolimod) capsules for the treatment of relapsing forms of multiple sclerosis (MS) in adult patients.
MS is a chronic, inflammatory, autoimmune disease of the central nervous system that disrupts communication between the brain and other parts of the body.
FDA approves novel treatment to target abnormality in sickle cell disease
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Today, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older.
"Today's approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this debilitating blood disorder," said Acting FDA Commissioner Adm. Brett P. Giroir, M.D.
FDA approves first treatment for inherited rare disease
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Today, the U.S. Food and Drug Administration granted approval to Givlaari (givosiran) for the treatment of adult patients with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood).
FDA approves first targeted therapy to treat patients with painful complication of sickle cell disease
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Today, the U.S. Food and Drug Administration approved Adakveo (crizanlizumab-tmca), a treatment to reduce the frequency of vaso-occlusive crisis - a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells - for patients age 16 years and older.
FDA approves first therapy to treat patients with rare blood disorder
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Today the U.S. Food and Drug Administration granted approval to Reblozyl (luspatercept-aamt) for the treatment of anemia (lack of red blood cells) in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.
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- FDA approves first oral GLP-1 treatment for type 2 diabetes