UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD
XTalks
FEBRUARY 14, 2022
Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. It is an autosomal recessive disorder, which means that if both parents are carriers of the faulty gene that causes the disease, there is a one in four chance that their child will develop the disease.
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