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Pompe disease

FDA to expedite development of two Sanofi rare disease drugs

FDA to expedite development of two Sanofi rare disease drugs

Pharmaforum

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

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

Amicus Therapeutics

clinical trials

Avalglucosidase alfa

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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

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Amicus Announces Positive Results for AT-GAA in Pompe Disease

Amicus Therapeutics

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FDA kills off Amicus’ 2018 accelerated approval plan

FDA kills off Amicus’ 2018 accelerated approval plan

Fierce Biotech

Amicus Therapeutics

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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

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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

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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

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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

clinical trials

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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

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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

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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

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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

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

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Genzyme pumps up Pompe meds; Fabrazyme still short

Genzyme pumps up Pompe meds; Fabrazyme still short

Fierce Pharma

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BioMarin's Pompe disease drug gets orphan status

BioMarin's Pompe disease drug gets orphan status

Yahoo/AP

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Genzyme Jumps on FDA Settlement, Approval of Pompe Remedy

Genzyme Jumps on FDA Settlement, Approval of Pompe Remedy

Barron's

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Genzyme eyes FDA ruling on Pompe drug

Genzyme eyes FDA ruling on Pompe drug

Bizjournals.com

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Genzyme: Positive Development on the Lumizyme Front

Genzyme: Positive Development on the Lumizyme Front

Seeking Alpha

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