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CNN – Health

A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access

By Brenda Goodman, CNN (CNN) — A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, its manufacturer announced Wednesday, making it the world’s most expensive medicine. Lenmeldy was approved by the US Food and Drug Administration on Monday and is the first therapy for

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What to know about puberty blockers

By Kristen Rogers, CNN (CNN) — As England’s National Health Service has decided to stop prescribing puberty blockers to minors with gender dysphoria or gender incongruence, many people are asking what those are and why they’re such a hot-button issue. “Puberty blockers are sort of like a man-made hormone analogue, and basically what they do

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FDA approves first drug for common form of liver inflammation

By Katherine Dillinger and Jen Christensen, CNN (CNN) — The US Food and Drug Administration has approved the first medication for a common form of liver inflammation called nonalcoholic steatohepatitis, or NASH, the agency said Thursday. NASH — also known as metabolic dysfunction-associated steatohepatitis, or MASH — happens when the liver becomes inflamed because of excess fat

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