Persistent Effect of Arimoclomol in Patients with Niemann-Pick Disease Type C: 24-Month Results from an Open-Label Extension of a Pivotal Phase 2/3 Study

2021 ◽  
Author(s):  
Marc C. Patterson ◽  
Eugen Mengel ◽  
Rosalia M. Da Riol ◽  
Mireia Del Toro ◽  
Federica Deodato ◽  
...  
Keyword(s):  
Disease Type ◽  
Phase 2 ◽  
Open Label ◽  
Pivotal Phase ◽  
Persistent Effect ◽  
Niemann Pick Disease ◽  
Open Label Extension ◽  
Type C ◽  
Niemann Pick ◽  
Pick Disease

Persistent effect of arimoclomol in patients with Nuemann-Pick disease type C: 12-month results from an open-label extension of a pivotal phase 2/3 study

2021 ◽  
Vol 132 (2) ◽  
pp. S83
Author(s):  
Marc C. Patterson ◽  
Eugen Mengel ◽  
Rosalia M. Da Riol ◽  
Mireia Del Toro ◽  
Federica Deodato ◽  
...  
Keyword(s):  
Disease Type ◽  
Phase 2 ◽  
Open Label ◽  
Pivotal Phase ◽  
Persistent Effect ◽  
Open Label Extension ◽  
Type C ◽  
Pick Disease

ANALYSIS OF CHOLESTEROL AND OXYSTEROL LEVELS IN NIEMANN PICK DISEASE TYPE C MICE AND PATIENTS

2006 ◽  
Vol 37 (S 1) ◽  
Author(s):  
S Tay ◽  
X He ◽  
AM Jenner ◽  
BS Wong ◽  
WY Ong
Keyword(s):  
Disease Type ◽  
Niemann Pick Disease ◽  
Type C ◽  
Niemann Pick ◽  
Pick Disease
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