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FDA Alerts

FDA Alert
FDA
FDA Alert
FDA Approves First Nonsteroidal Treatment for Patients With Duchenne Muscular Dystrophy
04/18/2024

Anthony Calabro, MA

Anthony Calabro, MA
The FDA approved givinostat, a nonsteroidal oral medication, for treating patients 6 years of age or older with Duchenne muscular dystrophy.
04/18/2024
FDA Alert
sickle cell disease
FDA Alert
First Gene Therapies Approved to Treat Adult, Pediatric Patients With Sickle Cell Disease
03/19/2024

Jessica Ganga

Jessica Ganga
The FDA approved two gene therapies, the first of their kind, to treat patients aged 12 years and older with sickle cell disease.
03/19/2024
FDA ALERT
medication
FDA ALERT
FDA Approves First Treatment for Rare, Degenerative Disease
04/18/2023
Jessica Ganga
The FDA has approved a treatment for Friedreich ataxia after the results of randomized, placebo-controlled study indicate a strong effectiveness in treatment.
04/18/2023
Enzyme Replacement Therapy
Enzyme Replacement Therapy
First Enzyme Replacement Therapy to Treat Rare Genetic Disorder Approved
03/17/2023
Jessica Ganga
Following a multicenter study, the FDA has approved the first enzyme replacement therapy in the United States to treat alpha-mannosidosis.
03/17/2023
Medication
Medication
FDA Approves First Treatment for Acid Sphingomyelinase Deficiency
09/20/2022
The FDA approved an intravenous infusion medication to treat symptoms of this rare genetic disease that affects the liver, spleen, lung, and brain.
09/20/2022