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» Niemann-Pick disease
Niemann-Pick disease
Azafaros begins subject enrolment in Phase II RAINBOW trial of AZ-3102
Clinical Trials Arena
Fri, 06/9/23 - 10:11 am
Azafaros
AZ-3102
Niemann-Pick disease
GM2 gangliosidosis
clinical trials
Sanofi's rare disease drug Xenpozyme scores FDA approval after nods in Japan and Europe
Fierce Pharma
Wed, 08/31/22 - 10:23 pm
Sanofi
Xenpozyme
Niemann-Pick disease
FDA
Sanofi's $20B buyout of Genzyme pays off again with European OK for first Niemann-Pick drug
Endpoints
Fri, 05/20/22 - 10:30 am
Sanofi
Genzyme
M&A
Europe
Niemann-Pick disease
Xenpozyme
olipudase alfa
FDA rejection of rare disease drug leads Orphazyme to cut two thirds of staff
MedCity News
Tue, 06/29/21 - 10:55 am
Orphazyme
FDA
arimoclomal
Niemann-Pick disease
layoffs
FDA rejects Orphazyme's rare disease drug, continuing wild ride for Danish meme stock
Fierce Biotech
Fri, 06/18/21 - 10:17 am
FDA
Orphazyme
Niemann-Pick disease
arimoclomol
‘There’s so much uncertainty’: As Mallinckrodt sells rare disease drug, parents worry about access
Stat
Thu, 05/20/21 - 11:06 pm
Mallinckrodt
Niemann-Pick disease
IntraBio Expands Niemann-Pick Trial to ID Neuroprotective, Disease-Modifying Effects
BioSpace
Mon, 11/9/20 - 09:06 pm
IntraBio
clinical trials
IB1001
Niemann-Pick disease
Nearly two years after their narrow PhIII failure, Orphazyme gets speedy look at the FDA
Endpoints
Wed, 09/16/20 - 10:38 am
Orphazyme
FDA
clinical trials
arimoclomal
Niemann-Pick disease
Sanofi Sees Positive Results in Two Clinical Trials for Nieman-Pick Disease
BioSpace
Thu, 01/30/20 - 09:30 pm
Sanofi
Niemann-Pick disease
olipudase alfa
clinical trials
A star drug in Mallinckrodt’s $1.2B Sucampo buyout flops in pivotal trial
Endpoints
Wed, 11/7/18 - 12:31 pm
Mallinckrodt
Sucampo
Niemann-Pick disease
Here’s why Sucampo paid $200M for its PhIII Niemann-Pick drug
Endpoints
Fri, 08/11/17 - 11:21 am
Sucampo
Niemann-Pick disease
cyclodextrin
Sanofi Genzyme Begins Pivotal Phase 2/3 Trial of Olipudase Alfa for Adult Patients with Acid Sphingomyelinase Deficiency
Yahoo
Wed, 07/6/16 - 09:49 am
Sanofi Genzyme
clinical trials
olipudase alfa
Niemann-Pick disease
FDA grants breathrough therapy designation to Genzyme's Olipudase Alfa
Yahoo/Reuters
Thu, 06/4/15 - 11:51 am
Genzyme
FDA
olipudase alfa
Sanofi
Niemann-Pick disease