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 Drug Approval Decisions Expected in March 2025
The FDA reviews drug approval decisions that are expected in March 2025. The FDA is reviewing the application for bentracimab for use as a ticagrelor reversal agent in patients with uncontrolled bleeding or in those who require urgent surgery or invasive procedure.
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.
FDA Approves Mirum Pharmaceuticals Treatment for Lipid-Storage Disease
The Food and Drug Administration approved Mirum Pharmaceuticals' Ctexli to treat Cerebrotendinous Xanthoma, a very rare lipid-storage disease. The approval makes Ctexli, or chenopod, the first FDA-approved drug to treat CTX,
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
How the FDA's approval for food additives works, and why experts say a loophole allows companies to bypass it
Experts say that for decades, a legal loophole has allowed companies to include chemical additives that could be potentially ...
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Health Sector Shifts: From Drug Approvals to Supply Disruptions
The current state of health services highlights crucial sectors facing disruptions and developments, from US foreign aid freezes affecting disease control supply chains, to the FDA's actions on new ...
FDA approves first rapid-acting insulin biosimilar for patients with diabetes
Aventis's Merilog (insulin-aspart-szjj) as a biosimilar to Novolog (insulin aspart) for adults and pediatric patients with ...
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, ...
Monthly Prescribing Reference3d
FDA Approves Vimkunya to Protect Against Chikungunya Virus
Vimkunya is supplied as an injectable suspension; the vaccine is administered intramuscularly as a single 0.8mL dose.
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The FDA Approves A New, Non-Opioid Painkiller
Here to help break down this new painkiller is Dr. Sean Mackey. He’s a Professor of Anesthesiology and Medicine at Stanford ...
News Medical on MSN3d
Vimseltinib gains FDA approval for treating tenosynovial giant cell tumor
The U.S. Food and Drug Administration (FDA) has approved vimseltinib (Romvimza TM ) for adult patients with a rare condition ...
OnePlus seeks FDA approval for Sleep Apnea Detection on its watch and takes on Apple in the process
OnePlus is hoping the OnePlus Watch 3 could get FDA approval for its new sleep apnea detection feature, bringing it one step closer to rivaling the best Apple Watches.