Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, ...
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Fetal therapy offers hope for unborn patients suffering from the rare genetic disease Hunter SyndromeBefore Ryann was born, a prenatal screening revealed he had a genetic disorder known as Hunter Syndrome or Mucopolysaccharidosis Type II. It is a rare genetic disorder that Queen's little brother ...
About DNL310 and Hunter Syndrome (MPS II) Hunter syndrome (MPS II) is a rare neurodegenerative lysosomal storage disease caused by mutations in the gene that encodes for the enzyme iduronate-2 ...
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