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Re: NPC1Parent post# 5565

Monday, 09/16/2019 7:23:36 PM

Monday, September 16, 2019 7:23:36 PM

Post# of 5675
Are you familiar with the work of Orphazyme?

(specifically, I would like to know what the term H1 & H2 mean)

and can you comment on this from their website:

Niemann-Pick disease Type C (NPC)
Data from the observational trial, NPC-001, showed a disease progression rate, which confirms the assumptions used to design the Phase II/III trial and candidate biomarkers were analyzed, confirming their potential use as disease biomarkers. Positive full data set announced in Q1 2019. Following EMA and FDA feedback mid-2019, Orphazyme expects to submit for filing in H1 2020, with potential approval in H2 2020.



https://www.orphazyme.com/clinical-programme
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