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Pompe disease
Amicus Puts Positive Spin on Failure of its Phase III Trial for Pompe Disease
Amicus Puts Positive Spin on Failure of its Phase III Trial for Pompe Disease
BioSpace
Amicus Therapeutics
clinical trials
Pompe disease
AT-GAA
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FDA to expedite development of two Sanofi rare disease drugs
FDA to expedite development of two Sanofi rare disease drugs
Pharmaforum
Sanofi
FDA
Rare Diseases
Pompe disease
Avalglucosidase alfa
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In anticipation of PhIII trial results, Amicus pares down old debt with $400 million loan
In anticipation of PhIII trial results, Amicus pares down old debt with $400 million loan
Endpoints
Amicus
Pompe disease
clinical trials
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Amicus shares surge as Sanofi struggles to raise a cheer for mixed pivotal data for a PhIII contender — and analysts blow a raspberry
Amicus shares surge as Sanofi struggles to raise a cheer for mixed pivotal data for a PhIII contender — and analysts blow a raspberry
Endpoints
Sanofi
Amicus Therapeutics
clinical trials
Pompe disease
Avalglucosidase alfa
Lumizyme
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Spark's CSO is wrapping up animal studies on a gene therapy for Pompe disease as new work in the field sees explosive growth
Spark's CSO is wrapping up animal studies on a gene therapy for Pompe disease as new work in the field sees explosive growth
Endpoints
Spark Therapeutics
Pompe disease
SPK-3006
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Amicus Announces Positive Results for AT-GAA in Pompe Disease
Amicus Therapeutics
AT-GAA
Pompe disease
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FDA kills off Amicus’ 2018 accelerated approval plan
FDA kills off Amicus’ 2018 accelerated approval plan
Fierce Biotech
FDA
Amicus Therapeutics
Pompe disease
AT-GAA
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Sarepta all-in on gene therapy pipeline, adding 3 CNS treatments in $30M Lacerta deal
Sarepta all-in on gene therapy pipeline, adding 3 CNS treatments in $30M Lacerta deal
Endpoints
Sarepta Therapeutics
CNS
Pompe disease
Lacerta
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Pompe Disease Patients on Amicus Drug Continue to Improve in Study
Pompe Disease Patients on Amicus Drug Continue to Improve in Study
Xconomy
Pompe disease
Amicus
ABT200
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Amicus’ latest positive update on Pompe drug hints at accelerated approval pitch
Amicus’ latest positive update on Pompe drug hints at accelerated approval pitch
Endpoints
Amicus Therapeutics
Pompe disease
ATB200/AT2221
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Sanofi Genzyme to begin late-stage trial of new drug for Pompe disease by summer
Sanofi Genzyme to begin late-stage trial of new drug for Pompe disease by summer
Bizjournals.com
Sanofi Genzyme
Pompe disease
clinical trials
NeoGAA
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BioMarin says it will continue studies of late-onset Pompe disease drug candidate BMN-701
BioMarin says it will continue studies of late-onset Pompe disease drug candidate BMN-701
Yahoo/AP
Biomarin
Pompe disease
BMN-701
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Amicus Therapeutics: One Biotech Firm Making Considerable Strides In Terms Of Pompe Disease
Amicus Therapeutics: One Biotech Firm Making Considerable Strides In Terms Of Pompe Disease
Seeking Alpha
Amicus Therapeutics
biotech
Pompe disease
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Amicus Theraputics announces positive results from Phase 2 Chaperone-ERT study
Amicus Theraputics announces positive results from Phase 2 Chaperone-ERT study
The Fly on the Wall
Amicus Therapeutics
AT2220
Pompe disease
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Will The Sanofi Pompe Paradigm Play Out With Neuralstem And Lou Gehrig's Disease?
Will The Sanofi Pompe Paradigm Play Out With Neuralstem And Lou Gehrig's Disease?
Seeking Alpha
Pompe disease
Sanofi
Neuralstem
Lou Gehrig's disease
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Piper Jaffray Boosts Target on BioMarin to $52 After Addition of BMN-701 to Model
Piper Jaffray Boosts Target on BioMarin to $52 After Addition of BMN-701 to Model
Street Insider
BMN-701
Biomarin
Pompe disease
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Genzyme pumps up Pompe meds; Fabrazyme still short
Genzyme pumps up Pompe meds; Fabrazyme still short
Fierce Pharma
Genzyme
Pompe disease
Fabrazyme
Myozyme
Lumizyme
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BioMarin's Pompe disease drug gets orphan status
BioMarin's Pompe disease drug gets orphan status
Yahoo/AP
Biomarin
Pompe disease
orphan drugs
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Genzyme Jumps on FDA Settlement, Approval of Pompe Remedy
Genzyme Jumps on FDA Settlement, Approval of Pompe Remedy
Barron's
Genzyme
FDA
Pompe disease
Lumizyme
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Genzyme eyes FDA ruling on Pompe drug
Genzyme eyes FDA ruling on Pompe drug
Bizjournals.com
FDA
Pompe disease
Genzyme
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