FDA, genetic disorder

US FDA approves Mirum Pharma's genetic disorder drug
The U.S. Food and Drug Administration approved Mirum Pharmaceuticals' genetic disorder drug on Friday, making it the first treatment for a rare condition that interferes with the body's ability to process cholesterol.
FDA approves new genetic disorder drug
The Food and Drug Administration (FDA) approved a new drug to treat a disorder causing the growth of noncancerous tumors on nerves throughout the body.  The federal agency said on Tuesday it
FDA Approves First Drug for Rare Lipid Storage Disorder
Chenodiol -- a naturally occurring human bile acid also known as chenodeoxycholic acid -- is the first FDA-approved drug indicated for the rare lipid storage disease and autosomal recessive genetic metabolic disorder, the agency said.
US FDA approves SpringWorks' genetic disorder drug
The U.S. Food and Drug Administration said on Tuesday it has approved SpringWorks Therapeutics' drug to treat a type of rare genetic disorder, which causes tumors to grow in the tissue that covers nerves throughout the body.
FDA Drug Approval Decisions Expected in March 2025
The Prescription Drug User Fee Act (PDUFA) date refers to the deadline set by the US Food and Drug Administration (FDA) for reviewing a New Drug Application (NDA) or Biologics License Application (BLA) and making a final decision on marketing approval.
FDA approves first treatment for rare lipid storage disease
The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease, in adults.According to a press release, Ctexli (chenodiol) is the first FDA-approved drug to treat CTX.
FDA Approved Journavx, a Non-Opioid Drug for Pain Management
On January 30, the U.S. Food and Drug Administration approved Journavx (suzetrigine) 50 milligram oral tablets, a first-in-class non-opioid
FDA approves Mirum treatment for cerebrotendinous xanthomatosis
The Food and Drug Administration approved Ctexli for the treatment of cerebrotendinous xanthomatosis in adults. “Ctexli is the first
Children with autism may see speech improvements with off-label prescription drug
An off-label prescription drug has been shown to help children with autism gain the ability to speak. Leucovorin is used for ...
New FDA-Approved Diabetes Drug Slashes Heart Attack and Stroke Risk
Sotagliflozin significantly reduces heart attacks and strokes in high-risk patients with diabetes and kidney disease, offering a new treatment option for cardiovascular protection. Sotagliflozin, a re ...
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1MIRM : FDA Approves Mirum Pharmaceuticals' Drug As First For Genetic Disorder...
FDA approves Mirum's Ctexli, the first treatment for cerebrotendinous xanthomatosis, a rare genetic disorder affecting fat metabolism. In a trial, Ctexli significantly reduced cholesterol ...