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Lead Product(s): Odiparcil
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 18, 2020
Details:
The results show that odiparcil was associated with decreased glycosaminoglycan (GAG) accumulation as well as increased GAG excretion, and highlight its distribution in MPS VI disease-relevant tissues and organs.
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Lead Product(s): Odiparcil
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 03, 2020
Details:
The Phase IIa clinical study results in MPS VI adult patients showed the capacity of odiparcil to improve key clinical parameters such as corneal clouding and cardiac and respiratory functions.
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