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New Drug Miplyffa Approved for Rare Niemann-Pick Disease Type 3

PLOS: DNA Science

The original approved was in 2003 to treat a type of Gaucher disease. But sometimes astute parents figure out what’s wrong before their doctors do. The post New Drug Miplyffa Approved for Rare Niemann-Pick Disease Type 3 appeared first on DNA Science. But like many new treatments for a rare disease, the price is high.

Disease 52
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Defense-Forward Biosecurity

Codon

Allison Berke makes the case for real-time DNA sequencing and AI tools to detect pathogens before they spread widely. Doctors in training are told that when they hear hoofbeats, they should think horses, not zebras; rare diseases are the exception, not the rule. After copying the DNA to form a big pool, each piece is sequenced.

DNA 102
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Welcome to Asimov Press

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Recombinant DNA technologies were invented in the 1970s. A first draft of the human genome was completed in 2003. When Ignaz Semmelweis suggested that doctors at an Austrian Hospital wash their hands between performing autopsies and delivering babies, the maternal mortality rate fell from around 25 percent to 1 percent.