Breaking News, Trials & Filings

JOT Submits IND to FDA

For Mucopolysaccharidosis Type-I (MPSI), utilizing their platform product, JOTROL

Jupiter Orphan Therapeutics has submitted its IND to the FDA on March 12, 2018 for Mucopolysaccharidosis Type-I (MPSI). This will be the first of several anticipated IND submissions in 2018 utilizing JOT’s platform product, JOTROL™.   Mucopolysaccharidosis I (MPSI, Hurler Syndrome, Hurler-Scheie Disease, Scheie Syndrome) is an autosomal recessive genetic disorder in which the alpha-L-iduronidase (IDUA) enzyme is greatly depleted or absent from the affected individual. The loss of I...

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